发明公开
- 专利标题: METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
- 专利标题(中): 治疗脑部疾病的方法和组合物
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申请号: EP12763234.7申请日: 2012-04-02
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公开(公告)号: EP2691529A1公开(公告)日: 2014-02-05
- 发明人: DAVIDSON, Beverly, L.
- 申请人: University of Iowa Research Foundation
- 申请人地址: 112 N. Capitol Street 6 Gilmore Hall Iowa City, IA 52242-5500 US
- 专利权人: University of Iowa Research Foundation
- 当前专利权人: University of Iowa Research Foundation
- 当前专利权人地址: 112 N. Capitol Street 6 Gilmore Hall Iowa City, IA 52242-5500 US
- 代理机构: Elbel, Michaela
- 优先权: US201161470460P 20110331
- 国际公布: WO2012135857 20121004
- 主分类号: C12N15/86
- IPC分类号: C12N15/86
摘要:
The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease.
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