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
Estimating the term…
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 transcarbamylasc 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.
Claims19
A recombinant viral vector comprising SEQ ID NO:
- 5.
A recombinant adeno-associated virus (rAAV) having an AAV capsid and packaged therein an expression cassette comprising at least one AAV ITR sequence, an engineered nucleic acid sequence encoding an ornithine transcarbamylase (OTC) protein, and expression control sequences which direct expression of the OTC protein in a liver cell, wherein the engineered nucleic acid sequence comprises a sequence at least 99% identical to SEQ ID NO:
- 5.
Dependencies are inferred from the claim text.