33.4 Gene therapy studies on hemophilia

In the year 1993 the stage I clinical trial of gene therapy was conducted by Lu D.R, et al ., for hemophilia B without much success. Subsequently, Kay M.A, et al ., in the year 2000 reported evidence of AAV mediated factor IX gene transfer and its expression in hemophilia B patients. Roth DA, et al ., in the year 2001 conducted a nonviral mediated factor VIII gene transfer in patients with severe hemophilia A . Powell JS, et al ., conducted phase 1 trial for severe hemophilia A in the year 2003 using a retroviral construct mediated FVIII gene transfer. The vector containing the construct was administered by peripheral intravenous infusion. Manno CS, et al ., in 2006 was able to successfully transduce hepatocytes by AAV mediated factor IX gene transfer. He studied and reported an elevated immune response hampering the gene therapy strategy used by him. Jiang H, et al ., in the year 2006 reported expression of factor IX in an individual with severe hemophilia B. In his strategy he used AAV mediated factor IX gene transfer which was administered to the skeletal muscle.