Gene Therapy for Hemophilia: Clinical Trials and Technical Tribulations

Abstract excerpt

As monogenic disorders, hemophilia A and B are compelling candidates for treatment with gene therapy. In hemophilia, a therapeutic benefit achieved by gene therapy should only require a modest increase in the endogenous factor level; response to treatment can be monitored easily; and there are relevant small and large animal models. The two main approaches aiming to restore factor VIII or factor IX production are...

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