Volume 2 (2015): Issue 2

Gene therapy for haemophilia: a very modern success story

Open Access | | Creative Commons Attribution NonCommercial NoDerivatives
Professor Amit Nathwani

Abstract

Abstract

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.

Article

View Full Article

References

  • 1. Morris J. Patient advocacy helps patients weigh up gene therapy trial risk/benefits. J Haem Pract 2015; 2(1): 6-8. doi: 10.17225/jhp.00040. Avaialble at http://www.haemjournal.com/external/articles/doi/10.17225/jhp.00040
  • 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: 10.1056/NEJMoa1108046.

PDF Download

Download PDF

Open in full-page viewer

Authors