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Molecular Treatment of Succinic Semialdehyde Dehydrogenase Deficiency with Messenger RNA
Unmet Need: Addressing the Imperative of Succinic Semialdehyde Dehydrogenase Deficiency
Succinic semialdehyde dehydrogenase deficiency (SSADHD) is a rare metabolic disorder caused by mutations in the ALDH5A1 gene. The enzyme plays a crucial role in the breakdown of gamma-aminobutyric acid (GABA). WSU Inventors used ALDH5A1-coding mRNA treatment method...
Published: 10/8/2024
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Inventor(s): Jean-Baptiste Roullet, K. Michael Gibson, Pranav Nawani
Keywords(s):
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