PASSINI, MARCO A.,STEWART, GREGORY R.,DODGE, JAMES
申请号：
PL04760694
公开号：
PL1620133T3
申请日:
2004.04.30
申请国别(地区):
PL
年份:
2016
代理人:
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.
