Gene therapy for haemophilia: a very modern success story

Open access


AAV-mediated gene therapy has the potential to be paradigm shifting as a treatment for haemophilia. A single administration of AAV vector can result in safe and consistent long-term expression of transgene (>5 years), reduction in spontaneous bleeding episodes, reduction in clotting factor usage and an improvement in quality of life. There is huge commercial interest in this approach, with the expectation that an AAV gene therapy product for haemophilia B will be licensed by 2020.

1. Morris J. Patient advocacy helps patients weigh up gene therapy trial risk/benefits. J Haem Pract 2015; 2(1): 6-8. doi:

2. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365(25): 2357-65. doi:

Journal Information


All Time Past Year Past 30 Days
Abstract Views 0 0 0
Full Text Views 43 43 4
PDF Downloads 36 36 3