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Gene therapy for haemophilia
Article first published online: 7 FEB 2008
© 2008 The Authors
British Journal of Haematology
Volume 140, Issue 5, pages 479–487, March 2008
How to Cite
Murphy, S. L. and High, K. A. (2008), Gene therapy for haemophilia. British Journal of Haematology, 140: 479–487. doi: 10.1111/j.1365-2141.2007.06942.x
- Issue published online: 7 FEB 2008
- Article first published online: 7 FEB 2008
- gene therapy;
- adeno-associated virus
The ultimate goal of gene therapy is the replacement of a defective gene sequence with a corrected version to eliminate disease for the lifetime of the patient. This challenging task is not yet accomplished, however significant progress is evident. An initial spate of clinical trials attempting the treatment of haemophilia with gene transfer primarily resulted in the demonstration of good safety profiles, but without efficacy. Subsequent reengineering of vector plasmids and delivery systems resulted in markedly improved outcomes in animal models of the disease. The most recent clinical trial for the treatment of haemophilia B with gene transfer showed transient achievement of efficacy in the highest dose cohort tested, but also exposed a previously hidden barrier to the future success of these treatments. The progress and problems of gene therapies for haemorrhagic disorders will be discussed. This review will concentrate on approaches in or near clinical application.