Rescuing Phenotypes in PTHS-Derived Brain Organoids with Alysson Muotri

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. [Show ID: 38516]

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[aniesbluff]

"Fix the brain which was previously damaged"... As a survivor of a ruptured brain aneurysm-(vascular hemorrhagic accident) this wonderful study sounds like a magnificent therapy to speed up healing our brains.
It took me 11 years to regain my memory and rebuild the person I was.

[Carlrod]

I strongly believe that the key factor, in the process, not only in this case, but in every process, associated with the neurological studies, is the signaling and every study, should strongly, focus, among other variables, in the origin of those signals

[lejlakaravdic]

I'm a mum of doughter ( two and half jear) diagnosed with pitt-hopkins syndrome, microdeletion. What about older children with pitt-hopkins and gene therapy? Or it work only postnatal or very early?