TREATMENT OF NEUROLOGICAL DISEASES USING ADENO-ASSOCIATED VIRUS (AAV) COMPRISING AAV-5 CAPSID PROTEINS

摘要

To provide methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord using an adeno-associated virus (AAV) gene therapy vector.SOLUTION: An adeno-associated virus (AAV) gene therapy vector for use as a medicament in a mammalian subject comprises AAV serotype 5 capsid proteins and a gene product of interest flanked by AAV ITRs, and is administered by intrathecal lumbar administration.SELECTED DRAWING: None