Article
Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.
The Journal of clinical investigation - 1 Jul 2006
Shi Qizhen, Wilcox David A, Fahs Scot A, Weiler Hartmut, Wells Clive W, Cooley Brian C, Desai Drashti, Morateck Patricia A, Gorski Jack, Montgomery Robert R
Abstract excerpt
Inhibitory immune response to exogenously infused factor VIII (FVIII) is a major complication in the treatment of hemophilia A. Generation of such inhibitors has the potential to disrupt gene therapy for hemophilia A. We explore what we believe to be a novel approach to overcome this shortcoming. Human B-domain-deleted FVIII (hBDDFVIII) was expressed under the control of the platelet-specific alphaIIb promoter in...
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