COMPOSITIONS USEFUL IN TREATMENT OF OTC DEFICENCY

摘要

Non-viral delivery systems comprising engineered human ornithine transcarbamylase (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 OTC deficiency (OTCD) patients by administering hOTC.