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 12.10.2025 Findings point to path forward for treatment of rare genetic disease News 12.08.2025 NIH Director Jay Bhattacharya visits Ó³»´«Ã½ to engage with scientists about the impact of federally-funded research News 11.24.2025 New AI model could speed rare disease diagnosis News 11.20.2025 FDA approves first cancer drug based on Ó³»´«Ã½ science All news
News 12.08.2025 NIH Director Jay Bhattacharya visits Ó³»´«Ã½ to engage with scientists about the impact of federally-funded research