Friday, May 7, 2021
Mitochondrial Health

Focus on Rare Disease: Prime time – gene therapy for rare disorders



RARE DISEASE DAY 2021
Focus on rare disease: Prime time — gene therapy for rare disorders
Wednesday, March 3
Virtual event hosted by the Broad Institute of MIT and Harvard

Sixth annual seminar showcasing rare disease research.

Agenda
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

For more information visit: Broad.io/RareDisease21

Chapters:

00:00 Welcome – Anna Greka
02:21 Polr3a Neonatal Progeroid Syndrome: A Medical Mystery – Leslie Macari
13:20 Keynote lecture – David Liu

Copyright Broad Institute, 2021. All rights reserved.

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