Gene therapy / en Wed, 13 May 26 14:42:12 -0400 Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study /news/precision-dna-editing-targets-root-cause-severe-childhood-epilepsy-preclinical-study <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Wed, 13 May 2026 18:42:12 +0000 tulrich@broadinstitute.org 5560466 at Single prime editing system could potentially treat multiple genetic diseases /news/single-prime-editing-system-could-potentially-treat-multiple-genetic-diseases <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Wed, 19 Nov 2025 17:01:34 +0000 kzusi@broadinstitute.org 5559721 at Prime editing treats childhood brain disease in mice /news/prime-editing-treats-childhood-brain-disease-mice <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Mon, 21 Jul 2025 15:37:05 +0000 Corie Lok 5559001 at Gene editing disrupts Huntington’s mutation in mice /news/gene-editing-disrupts-huntingtons-mutation-mice <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Tue, 27 May 2025 13:00:00 +0000 adicorat 5558521 at Evolved gene editor inserts entire genes in human cells /news/evolved-gene-editor-inserts-entire-genes-human-cells <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Thu, 15 May 2025 18:00:00 +0000 adicorat 5558566 at Gene editing extends lifespan in mouse model of prion disease /news/gene-editing-extends-lifespan-mouse-model-prion-disease <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Tue, 14 Jan 2025 10:00:00 +0000 adicorat 5558086 at Prime editing efficiently corrects cystic fibrosis mutation in human lung cells /news/prime-editing-efficiently-corrects-cystic-fibrosis-mutation-human-lung-cells <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Wed, 10 Jul 2024 09:00:00 +0000 adicorat 5557111 at A therapy candidate for fatal prion diseases turns off disease-causing gene /news/therapy-candidate-fatal-prion-diseases-turns-disease-causing-gene <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Thu, 27 Jun 2024 18:11:10 +0000 Corie Lok 5557021 at Improved prime editing system makes gene-sized edits in human cells at therapeutic levels /news/improved-prime-editing-system-makes-gene-sized-edits-human-cells-therapeutic-levels <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Mon, 10 Jun 2024 09:30:00 +0000 adicorat 5556946 at Prime editing shows proof of concept for treating sickle cell disease /news/prime-editing-shows-proof-concept-treating-sickle-cell-disease <span class="field field--name-title field--type-string field--label-hidden">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</span> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p> Identifying and Targeting Cancer Dependencies with Small Molecules</p> <p> <span style="display:none;">cell line profiling</span></p> </div> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Maria Nemchuk</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2017-05-04T11:41:42-04:00" title="Thursday, May 4, 2017 - 11:41" class="datetime">May 4, 2017</time> </span> <div class="field field--name-field-software-link field--type-link field--label-above"> <div class="field__label">Link</div> <div class="field__items"> <div class="field__item"><a href="ftp://caftpd.nci.nih.gov/pub/OCG-DCC/CTD2/Ó³»­´«Ã½/">CTD^2 Data Portal, Ó³»­´«Ã½ Datasets</a></div> </div> </div> <div class="field field--name-field-taxonomy-topic field--type-entity-reference field--label-above"> <div class="field__label">Topic</div> <div class="field__items"> <div class="field__item">cell line profiling</div> <div class="field__item">Downloads</div> <div class="field__item">Therapeutic Response</div> <div class="field__item">small molecules</div> <div class="field__item">Cancer</div> <div class="field__item">Portals</div> </div> </div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-above"> <div class="field__label">Ó³»­´«Ã½ tags</div> <div class="field__items"> <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/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/paul-clemons" hreflang="en">Paul Clemons</a></div> <div class="field__item"><a href="/broad-tags/stuart-schreiber" hreflang="en">Stuart Schreiber</a></div> </div> </div> Mon, 17 Apr 2023 15:53:54 +0000 kzusi@broadinstitute.org 1282061 at