发明名称 |
GENE THERAPY FOR LYSOSOMAL STORAGE DISEASES |
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. |
申请公布号 |
EP2158322(A1) |
申请公布日期 |
2010.03.03 |
申请号 |
EP20080756596 |
申请日期 |
2008.06.02 |
申请人 |
GENZYME CORPORATION |
发明人 |
DODGE, JAMES;CHENG, SENG, H. |
分类号 |
C12N15/86;A61K38/47;A61K48/00 |
主分类号 |
C12N15/86 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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