RNA interference for the treatment of gain-of-function disorders

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专利权人:

University of Massachusetts

发明人:

Neil Aronin,Phillip D. Zamore

申请号：

US16415826

公开号：

US11299734B2

申请日:

2019.05.17

申请国别(地区):

US

年份:

2022

代理人:

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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/