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DEPARTMENT OF HEALTH

GENE THERAPY RESEARCH

INVITATION TO TENDER

In response to the commitments made in the recently published White Paper ‘Our Inheritance, Our Future: Realising the potential of genetics in the NHS’, the Director of Research and Development now wishes to invite proposals for research on the translational mechanisms and service developments needed to move gene therapy for the inherited single gene disorders on from a research environment to the clinic. Proposals are also invited for research into the safety of gene therapy vectors and interventions.

Gene Therapy Research into Single Gene Disorders

Gene therapy offers the potential to cure or ameliorate genetic diseases by introducing working copies or modifications of defective genes into cells thereby correcting the inherited defect. Whilst some successes have been reported, progress has been relatively slow. Much of the current work in this area involves gene therapy for cancer and there is a funding gap for corresponding research on the inherited single gene disorders. This programme of commissioned research aims to fill this gap.

Research into the Safety of Gene Therapy

 Gene therapy has the potential for developing treatments for a wide range of diseases and disorders. However, concerns have been raised about the safety of this procedure. As with many new drugs or interventions, it has proven impossible to predict in advance the full range of potential adverse effects. With gene therapy these may be related to the therapeutic gene, the delivery vehicle and/or the route of administration. Research is therefore needed to monitor and assess the safety of different gene therapy vectors and interventions, particularly within the priority areas identified by the Gene Therapy Advisory Committee and the Committee on Safety of Medicines.

The detailed research brief for these commissioned programmes, application form and instructions are available on the Department of Health’s internet site : www.doh.gov.uk/research/callsforproposala.htm, or from Ms Beverley Walker, Research and Development Directorate, Skipton House, 80 London Road, London SE1 6HL or by email: [email protected].

Full proposals are to be submitted no later that 2.00pm on Friday 17 October 2003.