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

11/28/2022; 18 minutes

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. (#38516)

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