THROMBOSIS AND HEMOSTASIS Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment


A long-acting factor VIII (FVIII) as a replacement therapy for hemophilia A would significantly improve treatment options for patients with hemophilia A. To develop a FVIII with an extended circulating half-life, but without a reduction in activity, we have engineered 23 FVIII variants with introduced surface-exposed cysteines to which a polyethylene glycol… (More)


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