/ en Wed, 13 May 26 14:42:12 -0400 /news/precision-dna-editing-targets-root-cause-severe-childhood-epilepsy-preclinical-study <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Wed, 13 May 2026 18:42:12 +0000 tulrich@broadinstitute.org 5560466 at /news/clinical-trial-prion-disease-drug-candidate-begins-enrolling-participants <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Wed, 22 Apr 2026 13:35:55 +0000 jcolaros 5560386 at /news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Tue, 14 Apr 2026 13:00:00 +0000 jcolaros 5560371 at /rare-disease-day <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Thu, 05 Feb 2026 19:20:35 +0000 mnemchuk@broadinstitute.org 460961 at /news/new-ai-model-could-speed-rare-disease-diagnosis <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Mon, 24 Nov 2025 19:42:27 +0000 Corie Lok 5559751 at /news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Wed, 19 Nov 2025 17:01:34 +0000 kzusi@broadinstitute.org 5559721 at /news/prime-editing-treats-childhood-brain-disease-mice <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Mon, 21 Jul 2025 15:37:05 +0000 Corie Lok 5559001 at /news/count-me-launches-new-patient-partnered-research-project-translocation-renal-cell-carcinoma <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Tue, 11 Mar 2025 13:18:28 +0000 Corie Lok 5558201 at /news/gene-editing-extends-lifespan-mouse-model-prion-disease <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Tue, 14 Jan 2025 10:00:00 +0000 adicorat 5558086 at /news/clinical-genomicist-harnesses-team-based-science-help-rare-disease-patients <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" class="datetime">May 13, 2026</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Study in mice shows that base editing can correct the genetic mutation behind Dravet syndrome</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Roberto Molar Candanosa, The Jackson Laboratory </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2026-05-13T14:42:12-04:00" title="Wednesday, May 13, 2026 - 14:42" class="datetime">May 13, 2026</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1921px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=Y5XQYrsh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=hPA4NegH 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=62mDzYdo 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=u5RnRIrw 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L 1x" media="all and (max-width: 539px)" type="image/png" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/longstory/mouse-neurons%20NIH.jpeg_mo-BROAD00279830.png?itok=WpU5df3L" alt="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" title="Fluorescent microscopy image of two mouse neurons with branching nerve fibers spreading across a black background. Cell bodies glow pink and gold, with thin interconnected projections highlighted in yellow and magenta" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Christophe Leterrier, NeuroCyto Lab, INP, Marseille, France, via NIH BRAIN Initiative </div> <div class="media-caption__description"> Microscopy image of mouse neurons. </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/619/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/619/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/619/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-narrow paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><div class="clearfix" style="background-color:#f3f3f3;font-family:Open Sans;margin-bottom:2em;padding:2em;"> <h3>Highlights</h3> <ul> <li aria-level="1" data-list-item-id="e9fc78e6d510efd87ef443e22b42769e9">Dravet syndrome is a rare, incurable, and potentially deadly form of childhood epilepsy.</li> <li aria-level="1" data-list-item-id="e6deed81f1e23444820c71ce4fc1572b5">Scientists used base editing to correct a Dravet-associated mutation in the gene <em>SCN1A</em> in mice.</li> <li aria-level="1" data-list-item-id="ee63104c12aadab954561ce9951e74a16">The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</li> </ul> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/david-liu">David Liu</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute for Transformative Technologies in Healthcare</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=B9ePs0Mh 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=cN5T1axJ 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=gfYFXbBv 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_base-editing-Liu_ext-main_040826.png?itok=tRQbbPQi" alt="A letter C is penciled into a long DNA sequence. " title="A letter C is penciled into a long DNA sequence. " typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=P1HVVzXL 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=KdKAIdzp 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=QhzrPs_f 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/Gene%20Editing_David%20Liu_Main%20News%20Story%20image_v4_Option%202.png?h=a7d067a7&itok=3LTrPTP3" alt="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." title="A graphic displaying an oval containing a DNA double helix with several base pairs highlighted, connected to four icons representing patients." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Single prime editing system could potentially treat multiple genetic diseases</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-treats-childhood-brain-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=wehH4Ame 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=S3l31d6N 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=SoaktP-I 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/ATP1A3%20mutant%20iPSC%20neurons%20AHC%20model.jpg?h=dbc6d25a&itok=rwHlkGIZ" alt="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" title="Microscopy image showing human neurons in blue (indicating cell nuclei) and green (indicating mutated protein)" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-treats-childhood-brain-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing treats childhood brain disease in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=nUqDO0hj 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=8QwviADu 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=B7UQkdKU 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/NewsStory_Main_TNR_diseases_v3.png?itok=sL5HZC_R" alt="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." title="A graphic displaying two gray trains moving in opposite directions. One has a series of blue cars and the other has a series of blue cars with one green car in the middle. A lightning bolt is hitting the green car, which shows a green check mark." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/gene-editing-disrupts-huntingtons-mutation-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Gene editing disrupts Huntington’s mutation in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=aaqhDe9T 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=Gg0TBLag 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=eo_R7Q6w 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/News_Cystic_fibrosis_main-ext_0724_v2.png?h=d3e04ee7&itok=LwEAACqJ" alt="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." title="A graphic showing lungs inside a person with cystic fibrosis, and a double helix of DNA showing a three base pair correction." typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing efficiently corrects cystic fibrosis mutation in human lung cells</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/Gene_editors_mice_v4.png?itok=9simMt0F 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/Gene_editors_mice_v4.png?itok=vjppaTCV 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/Gene_editors_mice_v4.png?itok=OTLORMcm 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/Gene_editors_mice_v4.png?itok=vg-5yzMG" alt="Graphic showing particles delivering cargo to a mouse brain" title="Graphic showing particles delivering cargo to a mouse brain" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/researchers-engineer-vivo-delivery-system-prime-editing-partially-restoring-vision-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=_OtIoGoJ 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=oNPuC_bT 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=hKqIKriQ 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2023/24170591501_3be68fd5a6_o.jpg?itok=iAbN4a54" alt="Sickle cell disease" title="Sickle cell disease" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/prime-editing-shows-proof-concept-treating-sickle-cell-disease" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Prime editing shows proof of concept for treating sickle cell disease</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=5wJMT-Rt 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=IwzGNGES 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=ZY4Rhc3r 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/longstory/main_Fig3_Figure_3.png?h=b9df9531&itok=Kl1xKkPp" alt="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" title="Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/base-editing-treats-spinal-muscular-atrophy-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Base editing treats spinal muscular atrophy in mice</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=6FVq662l 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=03Y7sGv- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=2R67gsxG 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2021/NewbyLiu-SickleCell-MainImage.png?h=b44d6655&itok=cMctgO-v" alt="Zayna Sheikh, Ó³»­´«Ã½ Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/genetic-base-editing-treats-sickle-cell-disease-mice" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Genetic base editing treats sickle cell disease in mice</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.</p> <p>Published in <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>Science Translational Medicine</em></a>, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.</p> <p>"For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause," said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. "We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself."</p> <p>The work builds on a long-standing collaboration between JAX’s RDTC Vice President Cathleen (Cat) Lutz and <a href="/bios/david-liu">David Liu</a>, a core member of the Ó³»­´«Ã½, director of Ó³»­´«Ã½'s <a href="/merkin-institute-transformative-technologies-healthcare">Merkin Institute of Transformative Technologies in Healthcare</a>, and a pioneer in gene editing. The team has contributed to the growing momentum behind gene editing as a viable therapeutic strategy for rare diseases, a field that has seen landmark advances including the treatment of Baby KJ Muldoon in 2025. The latest breakthrough comes from their partnership with Ethan Goldberg, a pediatric neurologist at Children’s Hospital of Philadelphia and director of the Epilepsy Neurogenetics Initiative.</p> <p>Regulators are taking note of this momentum as well. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.</p> <h2>A new era of precision genetics</h2> <p>Dravet syndrome is a neurodevelopmental disorder that begins in infancy or early childhood, with symptoms including drug-resistant epilepsy, spontaneous and fever-triggered seizures, and significant developmental impairments. Patients face a high risk of sudden unexpected death. Yet current approved therapies often require repeated dosing or long-term intervention to treat symptoms. While the disease is rare by definition, an estimated 15,000–20,000 patients live with it in the United States today, Lutz said.</p> <p>The preclinical study focused on a specific Dravet-causing <em>SCN1A</em> variant called R613X. This mutation prevents cells from producing a full, functional Nav1.1 channel, which regulates neuron excitability. This creates an electrophysiological imbalance in certain brain cells, where neurons don’t work properly, making the brain overexcitable and prone to seizures. </p> <p>The researchers used adenine base editing, a precision gene editing approach that rewrites a single DNA letter without cutting both DNA strands, which preserves genomic integrity and reduces the risk of unintended edits. To correct the mutation in cells and in a mouse model of Dravet syndrome, the base editor was delivered via a single injection into the brain in very young mice, either on day one or day 12 after birth. </p> <p>The success of the approach is particularly significant, Lutz said, because it applies base editing to one of the most difficult settings for genetic medicine: a neurological disorder involving specialized inhibitory neurons distributed across the brain. </p> <p>In treated mice, the team corrected nearly 60 percent of the mutated DNA. Even with partial correction, almost all the gene’s expression appeared normal. That is because cells naturally help destroy defective messages from uncorrected genes. </p> <p>"The advantage here is that once you correct the gene, the cell's own regulatory systems take over again," Simon said. "You’re not managing a disease but restoring the biology that was always meant to be there."</p> <p>As predicted, the edit restored the function of the gene and prevented seizures. Unlike untreated mice, the mice treated at birth had a significant survival improvement. Mice treated on day 12 also benefited, with lasting protection into young adulthood and very low levels of unintended DNA changes or other adverse effects in the brain.</p> <p>"Most patients aren’t diagnosed at birth. They’re diagnosed after symptoms begin. So, showing that we can intervene later, at an age closer to real patients, is important," Simon continued. "There’s been a concern that once the brain develops, it may be too late to fix these problems. Our data suggest that’s not the case." </p> <p>Because Dravet syndrome patients often have their own unique mutations, the next step is figuring out how to tailor the approach across the many different versions of the disease and advancing delivery technologies for the clinic.</p> <h2>A new platform for genetic medicine</h2> <p>Last month, a JAX-Ó³»­´«Ã½ team reported using the same gene editing technology <a href="/news/base-editing-repairs-mutation-and-liver-function-mouse-model-zellweger-spectrum-disorder">to fix mutations in mice and human patient cells that cause an extremely rare and life-threatening genetic disease called Zellweger spectrum disorder</a>, which impairs liver function in early childhood. In 2025, that team also <a href="/news/prime-editing-treats-childhood-brain-disease-mice">fixed mutations in mice causing alternating hemiplegia of childhood</a>, a genetic disorder that causes life-threatening seizures in children. For that, they used an approach called prime editing, which rewrites (inserts, deletes, or replaces) short DNA sections instead of fixing single-letter errors.</p> <p>"We’re following a game plan built on years of work in the field, and this project showed how straightforward the process can be when all the pieces come together: the right model, testing the editing strategy, and connecting that all the way through to disease outcomes," Lutz said. "The long-term vision is to build a platform so robust and adaptable that correcting a new mutation becomes a matter of precision and speed rather than starting from scratch, ultimately expanding the reach of genetic medicine to diseases that today can only be managed."</p> <p>The team is focused on separating the unchanging components of the platform from those that need to be tailored to each disease—primarily the guide molecule that directs the gene editor to the precise location in the DNA that needs to be corrected. The goal is to establish the platform’s overall safety and efficacy so that adapting it to new mutations becomes increasingly practical.</p> <p>"This study gives us hope that base editing could be an effective approach for durably correcting the underlying cause of Dravet syndrome in patients," said Liu, who is also a Howard Hughes Medical Institute investigator and a professor at Harvard University. "It is also a compelling example of the benefits of working collaboratively across laboratories and institutions to integrate each other’s complementary expertise into the foundation for a future treatment for a devastating rare disease." </p> <p><em>Adapted from </em><a target="_blank" href="https://www.jax.org/news-and-insights/2026/may/precision-dna-editing-targets-root-cause-of-severe-childhood-epilepsy-in-preclinical-study"><em>a press release issued by The Jackson Laboratory</em></a><em>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>This work was supported by the National Institute of General Medical Sciences, the National Human Genome Research Institute, the National Institute of Neuological Disorders and Stroke, the Ó³»­´«Ã½ Chemical Biology and Therapeutics Science, the Howard Hughes Medical Institute, the Dravet Syndrome Foundation Research Grant and Postdoctoral Fellowship Grant, and The Brody Family Medical Trust Fund Fellowship in "Incurable Diseases" of The Philadelphia Foundation. </p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Nelson AT, Hill SF, Simon M, et al. <a target="_blank" href="https://www.science.org/doi/10.1126/scitranslmed.adx5999"><em>In vivo</em> adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model</a>. <em>Science Translational Medicine</em>. Online May 13, 2026. DOI: 10.1126/scitranslmed.adx5999.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/liu-lab" hreflang="en">Liu Lab</a></div> <div class="field__item"><a href="/broad-tags/merkin-institute-transformative-technologies-healthcare" hreflang="en">Merkin Institute for Transformative Technologies in Healthcare</a></div> <div class="field__item"><a href="/broad-tags/chemical-biology-and-therapeutics-science" hreflang="en">Chemical Biology and Therapeutics Science Program</a></div> <div class="field__item"><a href="/broad-tags/rare-disease" hreflang="en">Rare Disease</a></div> <div class="field__item"><a href="/broad-tags/gene-therapy" hreflang="en">Gene therapy</a></div> </div> </div> </div> </div> </div> Tue, 07 Jan 2025 14:00:00 +0000 adicorat 5557606 at