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Egenis · Research

Regulation of Gene Therapy in the UK (2005-2007)

Graciela Nowenstein

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Homepage: Graciela Nowenstein Email: nowenstein@rug.nl


The aim of this research is to explore gene therapists’ views in the United Kingdom about the institutions and/or structures that influence or have influenced developments in gene therapy. Special attention is given to the exceptional regulatory situation of the UK where gene therapy is submitted to the ethical scrutiny of a specially created body, the Gene Therapy Advisory Committee (GTAC). The influence of funding bodies and the media are also explored.


Data has mainly been generated via semi-structured interviews with gene therapists. In order to complete and contextualise the data thus gathered observation has been conducted in a research unit, at GTAC and at different events such as education and information days and conferences.


  • What is gene therapy? Answers to this question help to delineate GTAC’s sphere of legitimate competence according to interviewees. Gene therapy does not appear as a clearly defined area of biomedical research and practice. It is described as a diverse field that includes research on cancer, immunology, tissue regeneration, monogenic disorders, etc. In spite of this diversity, interviewees observe that there is enough commonality in the field to justify the existence of specific societies,journals, conferences and even a regulatory body.
  • The most basic definition of gene therapy interviewees give is the use of nucleic acids as drugs, or the introduction of some exogenous DNA with therapeutic aims.
  • The expected therapeutic effects should only concern the patient and not his or her progeny. The purposeful alteration of the germ line is thereby excluded.
  • Most show a moral dislike for the idea of modifications of the germ line. They also agree that it is not realistic to think about it happening soon. Yet, if germ line manipulation ever becomes technically possible, most hypothesise that it will be used as therapeutic technique.
  • What are the prospects of gene therapy? The promises that are made are less breath taking than in the early years. This view calls for a normalisation of the image of gene therapy: the basic knowledge is not yet there and time is needed to accumulate it; as in any other field of biomedical research, it is going to hurt and kill some of the subject-patients that will be submitted to it; is not going to be the magic bullet or the holy grail announced or expected; yet it is not an unnatural way of doing medicine.
  • Is the existence of GTAC justified? Within the existing context interviewees agree that GTAC is exerting a control that responds to scientific, social and political needs. These are related to: (i) the novelty of gene therapy as a therapeutic approach; (ii) the social and political sensitivity vis-à-vis this approach; (iii) the excessive enthusiasm and optimism of the 1990s.
  • Some interviewees note that GTAC is part of a more general phenomenon that concerns the entire biomedical sphere, namely the increasing amount of regulation to which biomedical research is submitted. This phenomenon has two important consequences for research: the bureaucratic burden on senior researchers has become greater and it is often necessary to find and fund regulatory expertise when it comes to submit clinical trial applications to ethical and technical approval.
  • However, within this hyper-regulated and bureaucratised context, the action of GTAC is described in positive terms: GTAC’s members have so far shown by their action an engagement in favour of gene therapy; they are well-informed and knowledge able; GTAC has adopted a case-by-case strategy that is seen as reasonable when it comes to give acceptable levels of risks.
  • The issue of regulation led interviewees to elaborate on their relationship to funding structures and the media, which is for many problematic. Some feel they need to sell their research to the media as well as to funding bodies to obtain money for research; this implies the risk of falling into communication traps, of being misinterpreted but also of producing non-realistic expectations in terms of future benefits and possible risks.

Further information

Graciela Nowenstein moved from Egenis in April 2007 to the University of Groningen in the Netherlands.