Update on clinical gene therapy for hemophilia

摘要: In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are currently in clinical development, gene therapy holds promise of a lasting cure with single drug administration. Near-to-complete correction A (factor VIII deficiency) and B IX have now been achieved patients by hepatic vivo transfer. Adeno-associated viral vectors different capsids engineered express high-level, some cases hyperactive, coagulation factors were employed. Patient data support sustained endogenous production clotting factor as result eliminates need infusion (or alternative drugs promote coagulation), may therefore ultimately also reduce treatment costs. However, mild liver toxicities observed receiving high vector doses. but not all instances, correlated T-cell response directed against capsid, prompting use immune suppression. addition, can be treated because preexisting immunity capsids. Nonetheless, studies animal models suggest approach used tolerance induction prevent or eliminate inhibitory antibodies factors. These form traditional protein replacement represent major complication treatment. The current review provides summary update on advances its continued development.