For central nervous system (CNS) applications, attempts are being made to deliver AAV gene therapy directly to the brain parenchyma or the cerebrospinal fluid (CSF) space by intra-cisterna magna ...

Maple syrup urine disease (MSUD) is a rare genetic inborn error of metabolism characterized by recurrent life-threatening neurologic crises and progressive brain ...

By testing different AAV serotypes—variants that target ... activity in vivo in the brain and tracing how brain circuits ...