Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.

ABSTRACT: Delivering drugs effectively to the central nervous system (CNS) is a major challenge in drug development, including adeno-associated virus (AAV) gene therapy. The cerebrospinal fluid (CSF) circulates through the ventricular system and the subarachnoid space, surrounding both the brain and spinal cord, making it an attractive target for CNS drug delivery. Here, we compare intra-cisterna magna (ICM) administration of four AAV serotypes, AAV1, AAV5, AAV9, and AAVDJ, carrying glucosylceramidase beta 1 (GBA1) cDNA encoding the lysosomal enzyme β-glucocerebrosidase (GCase), in non-human primates (NHPs). Mutations in GBA1 are causative for Gaucher's disease (GD) and the most important genetic risk factor for Parkinson's disease (PD). AAV delivery of GBA1 is a promisin

SUBMITTER: Okai T 

PROVIDER: S-EPMC12509745 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature