发明公开
EP2821085A2 RNA interference for the treatment of gain-of-function disorders
审中-公开
RNA-Interferenz zur Behandlung vonFunktionszunahmestörungen
- 专利标题： RNA interference for the treatment of gain-of-function disorders
- 专利标题（中）： RNA-Interferenz zur Behandlung vonFunktionszunahmestörungen
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申请号： EP14168602.2申请日： 2004-09-13
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公开(公告)号： EP2821085A2公开(公告)日： 2015-01-07
- 发明人: Aronin, Neil , Zamore, Phillip D
- 申请人： University of Massachusetts
- 申请人地址： 365 Plantation Street Worcester, MA 01605 US
- 专利权人： University of Massachusetts
- 当前专利权人： University of Massachusetts
- 当前专利权人地址： 365 Plantation Street Worcester, MA 01605 US
- 代理机构： Nash, David Allan
- 优先权： US502678P 20030912
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; C07H21/04 ; C12N15/11
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.
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