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publicationNumber US-9890365-B2
titleOfInvention Compositions useful in treatment of ornithine transcarbamylase (OTC) deficiency
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.
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