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• W2945798621 startingPage "743" @default.
• W2945798621 abstract "Abstract Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questions remain pertaining to its use in specific populations such as those with factor inhibitors, those with underlying liver disease, and pediatric patients. Here, the authors provide an update on viral vector and transgene improvements, review the results of recently published gene therapy clinical trials for hemophilia, and discuss the main challenges facing investigators in the field." @default.
• W2945798621 created "2019-05-29" @default.
• W2945798621 creator A5013407757 @default.
• W2945798621 creator A5033744481 @default.
• W2945798621 date "2019-05-16" @default.
• W2945798621 modified "2023-10-18" @default.
• W2945798621 title "Gene Therapy: Paving New Roads in the Treatment of Hemophilia" @default.
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• W2945798621 doi "https://doi.org/10.1055/s-0039-1688445" @default.
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• W2945798621 hasPublicationYear "2019" @default.
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