GNT0003 restores gene expression in Crigler Najjar syndrome

GNT0003 restores gene expression in Crigler Najjar syndrome

A single, intravenous infusion of GNT0003, an adeno-associated virus vector serotype 8, restored gene expression among patients with Crigler Najjar syndrome, according to a late breaker presentation at the International Liver Congress.“Crigler Najjar syndrome is a rare, inherited disorder of bilirubin metabolism due to mutations in uridine diphospho-glucuronate glucuronosyltransferase 1A1 (UGT1A1) causing deficit of UGT1A1 protein that conjugates bilirubin; if left untreated, this condition leads to severe neurological injury,” Lorenzo D’Antiga, MD, Pappa Giovanni XXIIIRead More

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