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Hemophilia

"Genetic Induction of Immune Tolerance to Human Clotting Factor VIII in a Mouse Model for Hemophilia A."

Published in Gene Therapy Weekly, June 29th, 1998

According to the authors' abstract of an article published in Proceedings of the National Academy of Sciences of the United States of America, "Patients with severe coagulation factor VIII deficiency require frequent infusions of human factor VIII (hFVIII) concentrates to treat life-threatening hemorrhages. Because these patients are immunologically hFVIII-naive, a significant treatment complication is the development of inhibitors or circulating alloantibodies against hFVIII, which bind the replaced glycoprotein, increase its plasma clearance, and inhibit its activity, preventing subsequent treatments from having a therapeutic effect. A genetic approach toward the induction of...

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