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

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 s...

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Detalles Bibliográficos
Autores principales:	Fong, Sylvia, Yates, Bridget, Sihn, Choong-Ryoul, Mattis, Aras N., Mitchell, Nina, Liu, Su, Russell, Chris B., Kim, Benjamin, Lawal, Adebayo, Rangarajan, Savita, Lester, Will, Bunting, Stuart, Pierce, Glenn F., Pasi, K. John, Wong, Wing Yen
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group US 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9018415/ https://www.ncbi.nlm.nih.gov/pubmed/35411075 http://dx.doi.org/10.1038/s41591-022-01751-0

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