发明名称 |
Gene therapy for neurometabolic disorders |
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. |
申请公布号 |
EP2269658(A1) |
申请公布日期 |
2011.01.05 |
申请号 |
EP20100179332 |
申请日期 |
2004.04.30 |
申请人 |
GENZYME CORPORATION |
发明人 |
PASSINI, MARCO, A.;STEWART, GREGORY R.;DODGE, JAMES |
分类号 |
A61K48/00;A61K38/46;C12N15/864 |
主分类号 |
A61K48/00 |
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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