New technologies in gene therapy for inducing immune tolerance in hemophilia A.

Authors: Borsotti C, Follenzi A Abstract INTRODUCTION: Conventional hemophilia treatment is based on repeated infusion of the missing clotting factor. This therapy is lifelong, expensive and can result in the formation of neutralizing antibodies, thus causing failure of the treatment and requiring higher doses of the replacement drug. Areas covered: Gene and cell therapies offer the advantage of providing a definitive and long-lasting correction of the mutated gene, promoting its physiological expression and preventing neutralizing antibody development. This review focuses on the most recent approaches that have been shown to prevent and even eradicate immune response towards the replaced factor. Expert Commentary: Despite the encouraging data demonstrated by ongoing clinical trial...

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