RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS
- 申请公布号：
- WO2005027980(A1)
- 申请号：
- WO2004US29968
- 申请日期：
- 2004.09.13
- 申请公布日期：
- 2005.03.31
- 申请人：
- UNIVERSITY OF MASSACHUSETTS;ARONIN, NEIL;ZAMORE, PHILLIP, D.
- 发明人：
- ARONIN, NEIL;ZAMORE, PHILLIP, D.
- 分类号：
- A61K48/00;C07H21/04;C12N15/113;(IPC1-7):A61K48/00
- 主分类号：
- A61K48/00
- 摘要：
- <p>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.</p>
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