DODGE James,DODGE James,CHENG Seng H.,CHENG Seng H.
申请号：
SI200831847
公开号：
SI2158322T1
申请日:
2008.06.02
申请国别(地区):
SI
年份:
2017
代理人:
摘要：
This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subjects brain. The transgene product may be any that is deficient in a lysosomal storage disease.
