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Focus on rare disease: Prime time — gene therapy for rare disorders (2021)

Welcome
Anna Greka, Broad Institute, 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, Broad Institute, Harvard University, HHMI 
 
Q&A panel
Moderator: Anna Greka, Chair of Focus on Rare Disease
Panelists: David Liu, Leslie Macari