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8:00am – 8:30am
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BREAKFAST
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8:30am - 8:40am
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WELCOME REMARKS
Daniel Geschwind, MD, PhD
Distinguished Professor and Associate Vice Chancellor, Precision Health
UCLA
Steven M Dubinett, MD
Interim Dean, David Geffen School of Medicine
UCLA
Dr. Allen and Charlotte Ginsburg
Honored Guests
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8:45am - 9:20am
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Hematopoietic Stem Cell Gene Therapy-Add it or Edit?
Donald Kohn, MD
Distinguished Professor of Microbiology, Immunology and Molecular Genetics
UCLA
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9:25am - 10:00am
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Engineering Next-Generation CAR-T Cells for Cancer Immunotherapy
Yvonne Chen, MD, PhD
Associate Professor of Microbiology, Immunology, and Molecular Genetics
UCLA
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10:00am - 10:15am
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BREAK
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10:15am - 11:00am
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PLENARY SPEAKER
Base Editing and Prime Editing: Precision Gene Correction Without Double-Strand DNA Breaks
David Liu, PhD
Professor and Director of the Merkin Institute of Transformative Technologies in Healthcare
Broad Institute
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11:00am - 12:00pm
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KEYNOTE SPEAKER
CRISPR Cures: Genome Editing as a New Biomedical Frontier
Jennifer Doudna, PhD
Li Ka Shing Chancellor's Chair in Biomedical and Health Sciences
Professor, Depts. of Molecular & Cell Biology and Chemistry
UC Berkeley
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12:00pm - 12:15pm
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ANNOUNCEMENT: 2022 GINSBURG FELLOW
Dr. Dan Geschwind, MD, PhD and Dr. Allen Ginsburg, MD
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12:15pm - 1:00pm
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LUNCH
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1:00pm - 1:30pm
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LIGHTNING ROUND: Trainee Presentations (3)
- Person 1: Kelsey Jarrett, PhD - Manipulating Hepatic Bile Acid Production using Genome Editing to Modulate Cardio-Metabolic Disease Risk
- Person 2: George Chen, PhD - Targeting enhancers of autism risk genes CHD8 and SCN2A using CRISPR-A corrects mutant gene expression and phenotypes in developing brain organoids
- Person 3: Grace McAuley - Base Editing of Hematopoietic Stem Cells Rescues T-Cell Development for CD3 Severe
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1:35pm - 2:10pm
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Fetal Therapy for Single-Gene Disorders
Tippi C. Mackenzie, MD
Professor and Director of the Eli and Edythe Broad Institute for Regenerative Medicine
UCSF
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2:15pm - 2:50pm
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Ethics and Governance of Human Genome Editing
R. Alta Charo, JD
Professor Emerita of Law & Bioethics
University of Wisconsin – Madison
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2:50pm - 3:00pm
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BREAK
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3:00pm - 3:20pm
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Disrupting Duchenne: Advancing Precision Genetic Medicine through Science, Policy, and Practice
Diane Berry, PhD
Senior Vice President, Global Policy, Government & Patient Affairs
Sarepta Therapeutics
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3:20pm - 3:40pm
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Development of Treatments for Rare Diseases
Emil D. Kakkis, MD, PhD
Chief Executive Officer, President and Director
Ultragenyx
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3:40pm - 4:00pm
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FDA’s Efforts to Advance Gene Therapy
Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research
U.S. Food and Drug Administration
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4:00pm - 4:10pm
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BREAK
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4:10pm - 4:50pm
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Meet the Experts: Frontiers in Ethical and Regulatory Perspectives
Panelists: R. Alta Charo, JD, Diane Berry, PhD, Emil D. Kakkis, MD, PhD, Peter Marks, MD, PhD
Moderator: Carrie Miceli, PhD, Professor of Microbiology Immunology and Molecular Genetics, UCLA
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4:50pm - 5:00pm
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CLOSING REMARKS
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5:00pm - 6:30pm
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WINE RECEPTION
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Home Accommodations Event Schedule 
