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:

Lili Wang,James M. Wilson

申请号：

US15861427

公开号：

US20180135030A1

申请日:

2018.01.03

申请国别(地区):

US

年份:

2018

代理人:

摘要：

Non-viral delivery systems 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 deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or 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/