Crigler-Najjar syndrome (CNS) and Gilbert syndrome (GS) are rare autosomal recessive disorders causing unconjugated hyperbilirubinemia due to reduced UGT1A1 enzyme activity, with CNS type 2 (CNS2) ...
PARIS--(BUSINESS WIRE)--Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy ...
Gene therapy for Crigler-Najjar syndrome appeared promising in patients treated at a higher dose, a phase I/II trial showed. A single infusion of GNT0003 -- an adeno-associated virus serotype 8 vector ...
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