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 08.08.2025 Blood-based test provides new diagnostic approach for multiple myeloma News 08.06.2025 Scientists develop technology that brings new precision to genome editing News 08.06.2025 Breathing low-oxygen air slows Parkinson’s progression in mice News 08.01.2025 Genomics points to potential targeted treatment options for pediatric gliomas All news