Tolerance induction by viral in vivo gene transfer
E Dobrzynski, RW Herzog - Clinical medicine & research, 2005 - Marshfield Clinic
Treatment of genetic disease by protein or gene replacement therapy is hampered by
immune responses to the therapeutic protein. An excellent example is formation of inhibitory
antibodies to coagulation factors in treatment of the X-linked bleeding disorder hemophilia.
Experiments in murine and canine models of hemophilia B (deficiency in factor IX) have
demonstrated sustained therapeutic levels of factor IX transgene expression following
hepatic adeno-associated viral gene transfer in animals with deletion and nonsense …
immune responses to the therapeutic protein. An excellent example is formation of inhibitory
antibodies to coagulation factors in treatment of the X-linked bleeding disorder hemophilia.
Experiments in murine and canine models of hemophilia B (deficiency in factor IX) have
demonstrated sustained therapeutic levels of factor IX transgene expression following
hepatic adeno-associated viral gene transfer in animals with deletion and nonsense …