RettEd: MECP2 and Rett Syndrome: How Do Your Child's Genes Fit into Gene Therapies?
Автор: International Rett Syndrome Foundation
Загружено: 2025-02-05
Просмотров: 770
Join the International Rett Syndrome Foundation (IRSF) and expert speakers for this RettEd Webinar to explore the foundational principles of the genetics of Rett syndrome, including a review of the basic biology processes like transcription and translation that underly genetic-based treatments. We'll delve into cutting-edge therapeutic genetic approaches, such as RNA therapeutics, gene therapy, and gene editing, and will cover delivery methods and potential side effects. Learn about how outcomes are monitored and optimized.
This session is designed to provide a comprehensive overview of genetic therapeutic advancements for families and caregivers.
Guest Speakers from Children's Hospital of Philadelphia:
-Eric Marsh, MD, PhD
-Holly Dubbs, MS, LCGC
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