COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY

Abstract: the present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mrna encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the otc deficiency is reduced.

Inventor(s): Kim Askew, Jou-Ku Chung, Frank DeRosa, Michael Heartlein, Shrirang Karve, Thomas McCauley, Lianne Smith, Ann J. Barbier

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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