Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
AssigneeThe Trustees of the University of Pennsylvania
InventorsLili Wang, James M. Wilson
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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.
Claims15
A composition comprising a non-viral carrier and a nucleic acid molecule comprising an engineered sequence encoding human ornithine transcarbamylase (hOTCase) and expression control sequences which direct expression of hOTCase in a liver cell, wherein the hOTC nucleic acid sequence is less than 80% identical to the wild-type hOTC sequence over at least the mature hOTC sequence of SEQ ID NO:
- 1, wherein said hOTC nucleic acid sequence is selected from the nucleic acid sequence comprising SEQ ID NO:
- 5 or a nucleic acid sequence at least about 96% identical thereto, a nucleic acid sequence selected from SEQ ID NO:
- 9, or a nucleic acid sequence at least about 96% identical thereto, a nucleic acid sequence comprising SEQ ID NO:
- 11 or a nucleic acid sequence at least about 96% identical thereto, or a nucleic acid sequence comprising SEQ ID NO:
- 13 or a nucleic acid sequence at least about 96% identical thereto.
Dependencies are inferred from the claim text.