Rescue of Behavioral and Electrophysiological Phenotypes in a Pitt-Hopkins Syndrome Mouse Model by Genetic Restoration of Tcf4 Expression

Investigators performed proof-of-concept viral gene therapy experiments using a conditional Tcf4 mouse model of Pitt-Hopkins syndrome and found that postnatally reinstating Tcf4 expression in neurons improved anxiety-like behavior, activity levels, innate behaviors, and memory.