PASSINI MARCO A.,STEWART GREGORY R.,DODGE JAMES,PASSINI Marco A.,STEWART Gregory R.,DODGE James
申请号:
SI200432298
公开号:
SI1620133T1
申请日:
2004.04.30
申请国别(地区):
SI
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.