Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency

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专利权人:

The Trustees of the University of Pennsylvania

发明人:

Wang, Lili,Wilson, James M.

申请号：

AU2015229750

公开号：

AU2015229750A1

申请日:

2015.03.09

申请国别(地区):

AU

年份:

2016

代理人:

摘要：

Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis cirrhosis in OTCD patients by administering hOTC.

来源网站：

中国工程科技知识中心

来源网址：

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