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 05.07.2025 How the Novo Nordisk Foundation Center at Ó³»´«Ã½ is using scalable technologies and AI to find the root cause of diabetes News 04.29.2025 Sentinel program among finalists for the $100 Million 100&Change competition News 04.16.2025 WhyIScience Q&A: How a chemist moved to the business side of science News 04.09.2025 Potential Alzheimer’s disease therapeutic target identified in brain immune cells All news
News 05.07.2025 How the Novo Nordisk Foundation Center at Ó³»´«Ã½ is using scalable technologies and AI to find the root cause of diabetes