发明名称 |
METHODS OF TREATING PARKINSON'S DISEASE USING VIRAL VECTORS |
摘要 |
Methods of delivering viral vectors, particularly recombinant adeno-associated virus (rAAV) virions, to the central nervous system (CNS) using convection enhanced delivery (CED) are provided. The rAAV virions include a nucleic acid sequence encoding a therapeutic polypeptide. The methods can be used for treating CNS disorders such as for treating Parkinson's Disease. |
申请公布号 |
US2016256534(A1) |
申请公布日期 |
2016.09.08 |
申请号 |
US201615081116 |
申请日期 |
2016.03.25 |
申请人 |
Genzyme Corporation ;The Regents of the University of California |
发明人 |
Bankiewicz Krystof;Cunningham Janet |
分类号 |
A61K38/51;A61K9/51;C12N7/00;A61K31/198;A61K51/04;A61K48/00 |
主分类号 |
A61K38/51 |
代理机构 |
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代理人 |
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主权项 |
1. A method for delivering recombinant AAV virions to a subject, comprising administering via convection-enhanced delivery (CED) said rAAV virions into the CNS of the subject, wherein said rAAV virions comprise a nucleic acid sequence encoding a therapeutic polypeptide. |
地址 |
Framingham MA US |