Open Access

Gene therapy for haemophilia: a very modern success story

   | Apr 21, 2018


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.

Publication timeframe:
Volume Open
Journal Subjects:
Medicine, Basic Medical Science, other, Clinical Medicine, Pharmacy, Pharmacology