Gene Therapy: Molecular Engineering of Factor VIII and Factor IX

Gene therapy for hemophilia?

“…Ironically, the parallel research in clotting factor concentrate (CFC) infusion therapies (which, of course, has permitted this to be a ‘non‐desperate’ genetic disease population) also provides a cogent scientific rationale for continuing to pursue gene transfer strategies for hemophilia A and B. Research efforts to improve biosynthetic efficiency, to enhance specific activity [5], to improve protein stability [6], to reduce immunogenicity [7], and to prolong protein intravascular survival [8] for infused CFC may be readily adaptable for producing an enhanced transgene for factor VIII or IX gene transfer, obviating some of the risk associated with having to administer very high doses of transgene. Particularly for gene transfer using viral vectors, this may substantially increase the likelihood of achieving therapeutic in vivo protein levels while concomitantly reducing the vector‐associated toxicity risk.…”