GENE THERAPY FOR TUBEROUS SCLEROSIS

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专利权人:

The General Hospital Corporation

发明人:

Xandra BREAKEFIELD,Casey MAGUIRE,Shilpa PRABHAKAR,David YELLEN

申请号：

US16613907

公开号：

US20200079824A1

申请日:

2018.05.17

申请国别(地区):

US

年份:

2020

代理人:

摘要：

The invention provides compositions and methods for treating tuberous sclerosis complex (TSC). In particular, provided are condensed tuberins (cTuberins), cTuberin nucleic acids, and recombinant adeno-associated viruses (rAAVs) carrying a cTuberin nucleic acid for treating a patient with TSC.

来源网站：

中国工程科技知识中心

来源网址：

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