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Hemophilia clinical gene therapy: brief review
Christopher E Walsh1, Katherine M Batt
1Mount Sinai School of Medicine, New York City, NY 10029, USA. Christopher-e.walsh@msnyuhealth.org
Abstract:
Genetic correction of hemophilia A and B was long considered amenable to the available gene transfer technologies. This assumption has come to fruition with the recent results of a phase I/II trial for hemophilia B. Here we review the clinical application of gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer science and technology. This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias.
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