Shibi B. Likhite

This study uses AAV9-mediated PIGA gene replacement to rescue PIGA deficiency phenotypes in mice, improving survival, brain structure, and neurological function.

This study shows that juvenile AAV9-MCT8 gene therapy improves neurological and behavioral deficits in an AHDS mouse model by restoring TH levels and gene expression.

This study shows that AAV9 gene therapy in a mouse model of CLN8-Batten disease improves lifespan, reduces pathology, and reverses behavioral issues.