Breadcrumb Home Videos Focus on rare disease: Prime time — gene therapy for rare disorders (2021) Welcome Anna Greka, Ó³»´«Ã½, Brigham and Women’s Hospital, Harvard Medical School Polr3a Neonatal Progeroid Syndrome: A Medical Mystery Leslie Macari, parent of a rare disease patient Keynote Lecture Base Editing and Prime Editing: Genome Editing Without Double-Strand Breaks David Liu, Ó³»´«Ã½, Harvard University, HHMI Q&A panel Moderator: Anna Greka, Chair of Focus on Rare Disease Panelists: David Liu, Leslie Macari Latest news News 06.15.2026 Scientists improve nearly every aspect of prime editing, moving it closer to treating more genetic diseases News 05.13.2026 Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study News 05.01.2026 Single-molecule tracker illuminates workings of cancer-related proteins News 04.22.2026 Clinical trial of a prion disease drug candidate begins enrolling participants All news
News 06.15.2026 Scientists improve nearly every aspect of prime editing, moving it closer to treating more genetic diseases
News 05.13.2026 Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study