Transgenic Targeting of Human FVIII Expression to Endothelial Cells Corrects the Murine Hemophilia A Phenotype and Re-Establishes a Releasable Pool of FVIII Together with VWF.
Abstract:The deficiency of Factor VIII (FVIII) leads to hemophilia A, a severe X-linked bleeding disorder. To date, it is still unknown with certainty where FVIII is synthesized and how the storage pool of desmopressin acetate (DDAVP)-releasable FVIII is established. In vitro studies performed by our laboratory have shown that FVIII will store with VWF in endothelial cells if FVIII expression is induced by transfection or transduction. This FVIII is released by agonist stimulation. Furthermore, our studies have demonst… Show more
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