Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Finn, Jonathan D.; Ozelo, Margareth C.; Sabatino, Denise E.; Franck, Helen W. G.; Merricks, Elizabeth P.; Crudele, Julie M.; Zhou, Shangzhen; Kazazian, Haig H.; Lillicrap, David; Nichols, Timothy C.; Arruda, Valder R.

doi:10.1182/blood-2010-06-288001

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Inhibitory antibodies to factor VIII (FVIII) are a major complication in the treatment of hemophilia A, affecting approximately 20% to 30% of patients. Current treatment for inhibitors is based on long-term, daily injections of large amounts of FVIII protein. Liver-directed gene therapy has been …