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 01.23.2026 AI generates short DNA sequences that show promise for gene therapies News 01.16.2026 Scientists develop molecules that may treat Crohn’s disease News 01.07.2026 Researchers uncover molecular roots of fibrosis or tissue scarring in inflammatory bowel disease News 12.22.2025 High-fat diets make liver cells more likely to become cancerous All news
News 01.07.2026 Researchers uncover molecular roots of fibrosis or tissue scarring in inflammatory bowel disease