Use of lysosomal hydrolase-containing aav vectors for preparation of medicaments for treating lysosomal storage disease

摘要

The disclosure pertains to methods for treating neurometabolic disorder including lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.