Messenger rna treatment for succinic semialdehyde dehydrogenase deficiency
AssigneeWashington State University
InventorsJean-Baptiste Roullet, Pranav Nawani
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Abstract
Provided herein is an mRNA therapy composition and method for use thereof to treat succinic semialdehyde dehydrogenase (SSADH) deficiency (SSADHD). ALDH5A1-coding mRNA is formulated and encapsulated in lipid nanoparticles (LNPs) or micelles, then administered to subjects with SSADHD to effect expression of ALDH5A1, restoring SSADH activity and resulting in neurometabolic improvement.
Claims14
A method for treating succinic semialdehyde dehydrogenase deficiency (SSADHD) in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of an mRNA coding for aldehyde dehydrogenase 5 family member A1 (ALDH5A1).
Dependencies are inferred from the claim text.