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]