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Article

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

1 Apr 2022

Abstract excerpt

Factor VIII gene transfer with a single intravenous infusion of valoctocogene roxaparvovec (AAV5-hFVIII-SQ) has demonstrated clinical benefits lasting 5 years to date in people with severe hemophilia A. Molecular mechanisms underlying sustained AAV5-hFVIII-SQ-derived FVIII expression have not been studied in humans. In a substudy of the phase 1/2 clinical trial ( NCT02576795 ), liver biopsy samples were collected...

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PMID: 35411075
DOI: 10.1038/s41591-022-01751-0

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