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Factor VIII

Mouse and nonhuman primate studies suggest an adeno-associated virus (AAV) encoding an optimized recombinant human factor VIII could help treat hemophilia A. In wild-type mice, tail vein injection of an AAV encoding the optimized factor VIII led to 10-fold higher expression of the factor than injection of vectors encoding a nonoptimized factor VIII variant. In a mouse model for hemophilia A, injection of the optimized vector decreased tail clip-induced blood loss compared with saline injection. In nonhuman primates, peripheral vein delivery of the vector resulted in stable expression of the factor. Next steps could include evaluating the vector in nonhuman primate models for hemophilia.

SciBX 6(9); doi:10.1038/scibx.2013.217
Published online March 7, 2013

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McIntosh, J. et al. Blood; published online Feb. 20, 2013;
Contact: Amit C. Nathwani, UCL Cancer Institute, University College London, London, U.K.