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.
In addition, the technique using newborn animals had the advantage of not prompting an immune response, which in many other cases eventually blocks the blood clotting activity of introduced factor VIII in hemophilic animals. Since treatment more than a year ago, the blood of the mice and dogs in this study has maintained a normal level of clotting factor activity, and the animals have had no incidents of bleeding. The study will be reported in the April 26 issue of the Proceedings of the National Academy of Sciences.
Gwen Ericson | EurekAlert!
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