发明名称 |
RNA interference for the treatment of gain-of-function disorders |
摘要 |
The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease. |
申请公布号 |
EP2821085(A2) |
申请公布日期 |
2015.01.07 |
申请号 |
EP20140168602 |
申请日期 |
2004.09.13 |
申请人 |
UNIVERSITY OF MASSACHUSETTS |
发明人 |
ARONIN, NEIL;ZAMORE, PHILLIP D |
分类号 |
A61K48/00;C07H21/04;C12N15/113 |
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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