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AssigneeRaNA Therapeutics, Inc.
InventorsFrank DeRosa, Michael Heartlein, Lianne Smith, Shrirang Karve
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The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
A method of treating ornithine transcarbamylase (OTC) deficiency, comprising administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbyamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset.
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A method of treating ornithine transcarbamylase (OTC) deficiency, comprising administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbyamylase (OTC) protein, wherein the mRNA comprises a polynucleotide sequence at least 80% identical to SEQ ID NO:
A pharmaceutical composition for treating ornithine transcarbamylase (OTC) deficiency, comprising an mRNA encoding an ornithine transcarbyamylase (OTC) protein and wherein the mRNA encoding the OTC protein comprises a polynucleotide sequence at least 70%, 75%, 80%, 85%, 90%, or 95% identical to SEQ ID NO:
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A pharmaceutical composition for treating ornithine transcarbamylase (OTC) deficiency, comprising an mRNA encoding an ornithine transcarbyamylase (OTC) protein, wherein the mRNA comprises a polynucleotide sequence at least 80% identical to SEQ ID NO:
Dependencies are inferred from the claim text.