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Gene therapy trials in the UK: is haemophilia a suitable 'model'?

Ludlam, C. A., Pasi, K. J., Collins, Peter William, Maggs, P. H. B. B., Cumming, A. M., Dolan, G., Fryer, A., Hill, F. G. H., Peake, I. R., Perry, D. and Smith, M. 2004. Gene therapy trials in the UK: is haemophilia a suitable 'model'? Clinical Medicine 4 (1) , pp. 54-56. 10.7861/clinmedicine.4-1-54

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Abstract

Gene therapy may be the next major advance for treatment of many diseases, and severe haemophilia (an inherited deficiency of coagulation factor VIII or IX) is a useful model. Progress in gene therapy has been slowed down following fatal multi-organ failure during an adenovirus vector trial for ornithine-transcarbamylase deficiency and two episodes of leukaemia in a retroviral vector trial for severe combined immunodeficiency trial. A small number of early haemophilia clinical trials are in progress or reported. This paper considers ethical and statutory issues related to gene therapy for severe haemophilia within the UK and how these can be addressed through a well-established national network of haemophilia centres. It is likely that these issues will be relevant to clinicians considering gene therapy for other diseases.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Systems Immunity Research Institute (SIURI)
Subjects: R Medicine > R Medicine (General)
Uncontrolled Keywords: adverse events; ethics; gene therapy; haemophilia; legislation
Publisher: Royal College of Physicians
ISSN: 1470-2118
Last Modified: 04 Jun 2017 06:32
URI: http://orca.cf.ac.uk/id/eprint/61623

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