Article

mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria

2022-10-21

Abstract excerpt

Argininosuccinate lyase (ASL) is a key enzyme integral to the hepatic urea cycle which is required for ammonia detoxification, and the citrulline-nitric oxide (NO) cycle for NO production. ASL deficient patients present with argininosuccinic aciduria (ASA), an inherited metabolic disease with hyperammonaemia and a chronic systemic phenotype with neurocognitive impairment and chronic liver disease. ASL deficiency a...

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Literature Corpus work: 52ebe51d-35b1-508b-9623-e38e79952a2e
DOI: 10.1101/2022.10.19.512931

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