4.4 Article

Neonatal GALT gene replacement offers metabolic and phenotypic correction through early adulthood in a rat model of classic galactosemia

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A pilot study of neonatalGALTgene replacement usingAAV9dramatically lowers galactose metabolites in blood, liver, and brain and minimizes cataracts inGALT-null rat pups

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Summary: Classic galactosemia is a rare metabolic disorder caused by GALT deficiency. Current standard treatment involves dietary restrictions, but long-term complications still occur. This study demonstrates the feasibility and effectiveness of GALT gene replacement therapy in restoring GALT levels in both liver and brain of GALT-null rats.

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