Rescuing Phenotypes in PTHS-Derived Brain Organoids with Alysson Muotri - Sanford Stem Cell Symposium 2022

Alysson Muotri discusses modeling Pitt-Hopkins syndrome (PTHS) using stem cells and brain organoids. He shares how rescuing TCF4 expression with CRISPR-mediated epigenetic induction of AAV vector delivery provides a gateway for targeted therapeutics for PTHS and related conditions. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 38516]

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Stem cell science is changing medicine and our understanding of human development. The Stem Cell Channel takes you into the labs where cutting edge-research takes place, introduces you to the scientists leading the way, and breaks down how these amazing discoveries impact everyday life and health.