发明申请
- 专利标题： COMPOSITIONS USEFUL IN TREATMENT OF OTC DEFICENCY
- 专利标题（中）： 组合物有用于治疗场外缺陷
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申请号： PCT/US2015019536申请日： 2015-03-09
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公开(公告)号： WO2015138357A3公开(公告)日： 2015-12-10
- 发明人: WANG LILI , WILSON JAMES M
- 申请人： UNIV PENNSYLVANIA
- 专利权人： UNIV PENNSYLVANIA
- 当前专利权人： UNIV PENNSYLVANIA
- 优先权： US201461950157 2014-03-09
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; A61K9/127 ; C07H21/04 ; C12N15/00
摘要：
Non-viral delivery systems comprising engineered human ornithine transcarbamylase (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 OTC deficiency (OTCD) patients by administering hOTC.
