Article
Comparison of platelet-derived and plasma factor VIII efficacy using a novel native whole blood thrombin generation assay.
Journal of thrombosis and haemostasis : JTH - 1 Dec 2015
Baumgartner C K, Zhang G, Kuether E L, Weiler H, Shi Q, Montgomery R R
Abstract excerpt
BACKGROUND: We have recently developed a successful gene therapy approach for hemophilia A in which factor VIII (FVIII) expression is targeted to platelets by the αIIb promoter. Levels of platelet-expressed FVIII (2bF8) achieved by gene therapy may vary between individuals due to differences in ex vivo transduction and gene expression efficiency. Accurate assays to evaluate 2bF8 efficacy are desirable. OBJECTIVE:...
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