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US 2020/0165593 A1Published application
Modified mRNA Encoding a Propionyl-CoA Carboxylase and Uses Thereof
AssigneeModernaTX, Inc.
InventorsSusan Sobolov-Jaynes, Romesh R. Subramanian, Judith L. Campagnari, Haren Vasavada, Zhiliang Cheng
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Abstract
Disclosed are methods and compositions for treating propionic academia based on mRNA therapy.
Claims11
A method of treating propionic acidemia in a patient in need thereof comprising administering to the patient a therapeutically effective amount of a composition comprising a modified mRNA molecule encoding a propionyl-CoA carboxylase polypeptide.
A pharmaceutical composition comprising a therapeutically effective amount of a modified mRNA molecule wherein the modified mRNA molecule encodes one or both of a propionyl-CoA carboxylase subunit.
Dependencies are inferred from the claim text.