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A laboratory mouse.Newborn mice and dogs with hemophilia A were restored to normal health through gene therapy developed by researchers at Washington University School of Medicine in St. Louis. The technique introduced into the animals' cells a gene that makes clotting factor VIII, a protein missing because of a genetic defect. "We are really pleased with the results, because the animals produced about 20 times more factor than has been achieved in prior attempts using gene therapy for hemophilia A in dogs," says senior author Katherine Parker Ponder, M.D., associate professor of medicine and of biochemistry and molecular biophysics.

