AAV TREATMENT OF HUNTINGTONS DISEASE

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专利权人:

UNIVERSITY OF MASSACHUSETTS

发明人:

MUELLER, CHRISTIAN,ARONIN, NEIL,PFISTER, EDITH L.

申请号：

CA3037929

公开号：

CA3037929A1

申请日:

2017.09.22

申请国别(地区):

CA

年份:

2018

代理人:

摘要：

Aspects of the disclosure relate to compositions and methods useful for treating Huntington s disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntingtons disease using the same.

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中国工程科技知识中心

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