Expression of human factor VIII under control of the platelet-specific αIIb promoter in megakaryocytic cell line as well as storage together with VWF
Q Shi, DA Wilcox, SA Fahs, PA Kroner… - Molecular genetics and …, 2003 - Elsevier
Hemophilia A, which results in defective or deficient factor VIII (FVIII) protein, is one of the
genetic diseases that has been addressed through gene therapy trials. FVIII synthesis does
not occur in normal megakaryocytes. In hemophilia patients who have inhibitors to FVIII
activity, megakaryocytes could be a protected site of FVIII synthesis and subsequent release.
Since von Willebrand factor (VWF) is a carrier protein for FVIII, we hypothesize that by
directing FVIII synthesis to megakaryocytes, it would traffick together with VWF to storage in …
genetic diseases that has been addressed through gene therapy trials. FVIII synthesis does
not occur in normal megakaryocytes. In hemophilia patients who have inhibitors to FVIII
activity, megakaryocytes could be a protected site of FVIII synthesis and subsequent release.
Since von Willebrand factor (VWF) is a carrier protein for FVIII, we hypothesize that by
directing FVIII synthesis to megakaryocytes, it would traffick together with VWF to storage in …