Immune tolerance induced by platelet-targeted Factor VIII gene therapy in hemophilia A mice is CD4 T cell-mediated

摘要

BackgroundImmune responses are a major concern in gene therapy. Our previous studies demonstrated that platelet-targeted FVIII (2bF8) gene therapy together with in vivo drug-selection of transduced cells can rescue the bleeding diathesis and induce immune tolerance in FVIII^null mice.