RNA INTERFERENCE TO TREAT GAIN-OF-FUNCTION DISORDERS

• 收藏
• 分享

专利权人:

University of Massachusetts

发明人:

ARONIN, Neil,Zamore, Phillip D

申请号：

DK14168602

公开号：

DK2821085T3

申请日:

2004.09.13

申请国别(地区):

DK

年份:

2020

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/