NeuroscienceMolecular TherapyPeer-reviewed
AAV9 gene therapy restores lifespan and treats pathological and behavioral abnormalities in a mouse model of CLN8-Batten disease
Tyler B. Johnson, Katherine A. White, Jon J. Brudvig…·Jill M. Weimer
This study shows that AAV9 gene therapy in a mouse model of CLN8-Batten disease improves lifespan, reduces pathology, and reverses behavioral issues.
39 citations