RNA INTERFERENCE FOR THE TREATMENT OF GAIN-OF-FUNCTION DISORDERS

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专利权人:

University of Massachusetts

发明人:

Aronin Neil,Zamore Phillip D.

申请号：

US201615226476

公开号：

US2017051283(A1)

申请日:

2016.08.02

申请国别(地区):

美国

年份:

2017

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/