4 Million Awarded for Gene therapy trials

13 December 2004

£3 million in funding for trials of gene therapy for three single gene disorders, Duchenne muscular dystrophy (DMD), haemophilia and childhood blindness. An additional £1 million has been allocated to fund research into the long-term safety of gene therapy techniques. This funding is part of the £50 million set aside by the government in the 2003 Genetics White Paper Our inheritance, our future - realising the potential of genetics in the NHS to incorporate new genetic technologies into the NHS. Gene therapy refers to the deliberate replacement, removal or introduction of genes (or other genetic material) into the somatic cells of an individual to prevent or treat disease. Announcing the allocation of funding, Health Secretary John Reid commented: Investment saves lives - that is why it is vital that we fund research into the latest cutting edge treatments such as gene therapy so that Britain remains at the forefront of medical research. (see press release).

The Muscular Dystrophy Campaign will receive £1.6 million for research at Imperial College London aimed at developing a 'molecular patch' (comprising anti-sense oligonucleotides) to replace part of the defective dystrophin gene, with the aim of restoring partial dystrophin production to affected children. It is hoped this would significantly moderate the severity of DMD in treated individuals; there is currently no treatment for DMD, and affected individuals usually suffer progressive disability followed by death in their early twenties. £500,000 will go to Oxford BioMedica, a spin-out company from the University of Oxford, for a trial of their treatment for Haemophilia A (an inherited blood-clotting disorder caused by a mutation in the gene for blood-clotting Factor VIII), and the remaining £900,000 will fund a trial of therapies for inherited childhood blindness led by Dr Robin Ali of the Institute of Ophthalmology and Institute of Child Health, University College London. These gene therapy trials will all be the first of their kind in the UK. The £1 million for research into safety issues has been allocated to four groups at the University of Glasgow, the Royal Free and University College London Medical School, Kings College London and University College London for research in areas including the safety of retroviral and adenovirus-associated vectors and hydrodynamic gene delivery.

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