Rescue of glutaric aciduria type I in mice by liver-directed therapies
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DOI: 10.1126/scitranslmed.adf4086
Abstract: Glutaric aciduria type I (GA-1) is an inborn error of metabolism with a severe neurological phenotype caused by the deficiency of glutaryl–coenzyme A dehydrogenase (GCDH), the last enzyme of lysine catabolism. Current literature suggests that… read more here.
Keywords: brain; aciduria type; glutaric aciduria; liver directed ... See more keywords