GENE THERAPY FOR TREATING CITRULLENEMIA

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专利权人:

The Trustees of the University of Pennsylvania

发明人:

James M. Wilson,Jenny Agnes Sidrane,Lili Wang

申请号：

US16478971

公开号：

US20190352668A1

申请日:

2018.02.01

申请国别(地区):

US

年份:

2019

代理人:

摘要：

Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).

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中国工程科技知识中心

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