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Although hemophilia A seems particularly suitable for gene therapy because even low amounts of plasma coagulation factor VIII (FVIII) provide a significant clinical benefit to the patients, the ideal target cell for recombinant FVIII expression and gene therapy approaches remains to be identified. In this study, we tested the capacity of cord blood-derived endothelial progenitor cells (CBECs) for FVIII expression on stable lentiviral transduction.

Sustained Expansion and Transgene Expression of Coagulation Factor VIII–Transduced Cord Blood–Derived Endothelial Progenitor Cells