Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response

Abstract excerpt

Gene therapy for the X-linked bleeding disorder hemophilia B'factor IX (FIX) deficiency'by means of in vivo gene transfer with adeno-associated viral (AAV) vectors has been in clinical trials for the past 16 years, cycling between partial successes in the clinic and further development in the laboratory. At the recent World Congress of the International Society on Thrombosis and Haemostasis (Toronto, Canada, June...

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