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Recombinant adeno-associated virus vectors encoding acid beta glucosidase for use in treating gaucher disease
专利权人:
GENZYME CORPORATION
发明人:
申请号:
IL25289817
公开号:
IL252898A
申请日:
2017.06.14
申请国别(地区):
IL
年份:
2019
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 subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
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中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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