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Fateme Babaha (Pathology & Molecular Medicine- Investigation of Hemophilia A gene therapy
Manage episode 452251855 series 3368682
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Hemophilia A (HA) is an X-linked bleeding disorder caused by a mutation in the factor 8 (F8) gene that codes for FVIII coagulation protein. FVIII is naturally synthesized in the liver and the mutation results in the loss of function of FVIII protein which is critical in the blood clotting cascades. HA occurs is 1 to 5000 male births and it is estimated that there are a total of 3000 patients with HA in Canada. Currently, HA patients are treated with replacement therapy of the deficient factor. However, this approach is transient because of the short half-life of recombinant or plasma derived FVIII and is insufficient due to the formation of anti-drug antibodies. The alternative approach which is curative and promises a long-lasting expression of FVIII protein is gene therapy.
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