Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice

Nguyen, Giang N.; Lindgren, Jonathan R.; Seleme, Maria C.; Kafle, Samita; Zander, Catherine B.; Zheng, X. Long; Sabatino, Denise E.

doi:10.1016/j.jtha.2023.04.012

Journal of Thrombosis and Haemostasis

2023

DOI: 10.1016/j.jtha.2023.04.012

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Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice

Giang N. Nguyen

Jonathan R. Lindgren

Maria C. Seleme

et al.

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Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice

Cited by 3 publications

References 31 publications

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Cellular stress and coagulation factor production: when more is not necessarily better

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice

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