Invention Grant
- Patent Title: Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
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Application No.: US15124620Application Date: 2015-03-09
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Publication No.: US09890365B2Publication Date: 2018-02-13
- Inventor: Lili Wang , James M. Wilson
- Applicant: The Trustees of the University of Pennsylvania
- Applicant Address: US PA Philadelphia
- Assignee: The Trustees of the University of Pennsylvania
- Current Assignee: The Trustees of the University of Pennsylvania
- Current Assignee Address: US PA Philadelphia
- Agency: Howson & Howson LLP
- International Application: PCT/US2015/019536 WO 20150309
- International Announcement: WO2015/138357 WO 20150917
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N9/10 ; C12N15/86
Abstract:
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.
Public/Granted literature
- US20170021037A1 COMPOSITIONS USEFUL IN TREATMENT OF ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY Public/Granted day:2017-01-26
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