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Factor IX

Mouse studies suggest lentivirus-mediated delivery of a hyperfunctional mutant factor IX could help treat hemophilia. In a mouse model of hemophilia B, low doses of lentiviral vectors encoding an optimized hyperfunctional R338L factor IX mutant led to a
15-fold gain in factor IX activity, and greater factor IX protein levels and less blood loss in a tail-clipping assay than vectors encoding a wild-type or nonoptimized factor IX. None of the tested vectors induced immunogenicity or antibodies against factor IX. Next steps include testing the strategy in larger animal models, including in canines.
At least six companies have recombinant
factor IX or delivery methods for the coagulation factor in development stages ranging from preclinical to marketed to treat hemophilia.

SciBX 5(42); doi:10.1038/scibx.2012.1113
Published online Oct. 25, 2012

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Cantore, A. et al. Blood; published online Oct. 4, 2012;
Contact: Thierry VandenDriessche, Free University of Brussels, Brussels, Belgium