Restoring lost gene activity prevents many disease signs in an animal model of the rare, single-gene neurodevelopmental condition. The UNC Neuroscience Center lab of Ben Philpot, PhD, led this research.

Article:

https://elifesciences.org/articles/72290

UNC press release:

https://news.unchealthcare.org/2022/05/gene-therapy-could-treat-pitt-hopkins-syndrome-proof-of-concept-study-suggests/