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, Jacob T. Cain, Logan M. Langin, Melissa A. Pratt, Clarissa D. Booth, Derek J. Timm, Samantha S. Davis, Brandon L. Meyerink, Shibi B. Likhite, Kathrin Meyer, 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.
Molecular Therapy · Sep 24, 2020Peer-reviewedNeuroscience