COMPOSITION COMPRISING AN ADENO-ASSOCIATED VIRUS VECTOR HAVING A SEROTYPE 1 CAPSID FOR USE IN TREATING LYSOSOMAL STORAGE DISEASES OR ALZHEIMER'S DISEASE

摘要

The disclosure pertains to methods and compositions for treating disordersaffecting the central nervous system (CNS). These disorders includeneurometabolic disorders such as lysosomal storage diseases that affect thecentral nervous system, e.g., Niemann-Pick A disease. They also includedisorders such as Alzheimer's disease. The disclosed methods involvecontacting an axonal ending of a neuron with a composition containing hightiter AAV carrying a therapeutic transgene so that the AAV vector is axonallytransported in a retrograde fashion and transgene product is expresseddistally to the administration site.