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
Viral vectors 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 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.
Claims2
A recombinant viral vector comprising a nucleic acid sequence encoding human ornithine transcarbamylase (hOTC) and expression control sequences which direct expression of hOTC in a liver cell, wherein the hOTC nucleic acid sequence is less than 80% identical to the wild-type hOTC sequence over the mature sequence or full length hOTC of SEQ ID NO:
- 1, and expresses a functional hOTC, wherein said hOTC nucleic acid sequence is SEQ ID NO:
- 9 or a nucleic acid sequence at least 96 to 99% identical thereto.
A recombinant viral vector comprising a nucleic acid sequence encoding hOTC and expression control sequences which direct expression of hOTC in a liver cell, wherein the hOTC nucleic acid sequence expresses a functional hOTC, wherein said hOTC nucleic acid sequence is SEQ ID NO:
- 3.
Dependencies are inferred from the claim text.