Phenotypic correction and stable expression of factor VIII in hemophilia A mice by embryonic stem cell therapy
“Phenotypic correction and stable expression of factor VIII in hemophilia A mice by embryonic stem cell therapy”, vol. 12, pp. 1511-1521, 2013.,
Hereditary deficiency of factor VIII (FVIII) leads to hemophilia A, a severe X-linked bleeding disorder. Current therapies include fixed-dose FVIII prophylaxis, factor replacement therapy, and most recently, gene therapy. Prophylaxis and FVIII replacement therapies are limited by incomplete efficacy, high cost, restricted availability, and development of neutralizing antibodies in chronically treated individuals. Limited success has been obtained in preclinical trials using gene therapy for the treatment of hemophilia. Therefore, new options for therapy for hemophilia A are needed.