FMR1 gene therapy restores activity-driven inhibition and prevents audiogenic seizures in Fmr1-/y mice
Beatriz Maio, Aditi Singh, Ralph Hector, Kamal K.E. Gadalla, Jim Selfridge, Francesca Aria, Susana R. Louros, Stuart Cobb, Emily K. Osterweil
This study shows that AAV-FMR1 gene therapy restores auditory circuit function and prevents audiogenic seizures in Fmr1-/y mice by normalizing translation and excitatory/inhibitory balance in the inferior colliculus.
bioRxiv · Jun 16, 2026 · PreprintNeuroscience