Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.

ABSTRACT: Delivery of adeno-associated viral (AAV) vectors into the cerebrospinal fluid (CSF) can achieve gene transfer to cells throughout the brain and spinal cord, potentially making many neurological diseases tractable gene therapy targets. Identifying the optimal route of CSF access for intrathecal AAV delivery will be a critical step in translating this approach to clinical practice. We previously demonstrated that vector injection into the cisterna magna is a safe and effective method for intrathecal AAV delivery in nonhuman primates; however, this procedure is not commonly used in clinical practice. More routine methods of administration into the CSF are now being explored, including intracerebroventricular (ICV) injection and injection through a lumbar puncture. In this study, we compared I

SUBMITTER: Hinderer C 

PROVIDER: S-EPMC5770082 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature