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US 10,626,382 B2Granted
Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
AssigneeThe Trustees of the University of Pennsylvania
InventorsLili Wang, James M. Wilson
Life of this patent
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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.
Claims12
A composition comprising a nucleic acid molecule comprising an mRNA having a sequence of any one of SEQ ID NO:
- 10, a sequence which is 95% to 99% identical to SEQ ID NO:
- 10, SEQ ID NO:
- 11, or a sequence which is 95% to 99% identical to SEQ ID NO:
- 11, wherein the mRNA expresses a mature human ornithine transcarbamylase having a sequence of SEQ ID NO:
- 2.
2 more elements
Dependencies are inferred from the claim text.