Induction of factor VIII tolerance by hemophilia gene transfer to eradicate factor VIII inhibitors

Abstract excerpt

ABSTRACT: Patients with hemophilia A can develop antifactor antibodies to factor VIII. The incidence is ∼30%, and such patients suffer worse morbidity and mortality. The only proven method to eradicate these inhibitors is via immune tolerance induction therapy, which consists of infusing factor VIII concentrates at regular intervals. This approach is effective ∼65% of the time, leaving at least a third of...

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