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NHS behind world's first gene treatment for blindness

Published on 08/05/08 at 11:38pm

The world's first gene transplant to treat blindness, partly funded by the UK government, has successfully improved the night vision of one teenage patient.

The trial involved injecting healthy genes into the retinas of young people suffering from Leber's congenital amaurosis, an inherited condition which leads to progressive deterioration in vision and blindness.

Steven Howarth, aged 18, found the procedure had helped him see much better than before in low light conditions.

It is now hoped the treatment could be used to tackle a range of eye conditions, including inherited retinal diseases which affect one in 3,000 people, and even the common problem of age-related macular degeneration.

The research was pioneered by a team from the Moorfields Eye Hospital and the UCL Institute of Ophthalmology National Institute for Health Research (NIHR) Biomedical Research Centre, and was made possible by a £1 million grant from the Department of Health.

The gene treatment represents the first major outcome from the 12 biomedical research centres, which were set up in April last year by the NIHR and will receive £485 million over five years.

The breakthrough relied in part on the translational research - turning cutting-edge science into cures for patients - for which the centres were established in the first place.

Professor Sally Davies, director of R&D at the Department of Health, said: "This is fantastic work by the research team and shows how this country is now leading some of the most exciting health research in the world today."

"The success of this research has huge implications for sufferers of this condition," said public health minister Dawn Primarolo. "This kind of research is absolutely vital for the health and wellbeing of the nation."

Sir David Cooksey, author of the Chancellor's Cooksey Review, also praised the work. "When I published my review of health research funding, I emphasised that we do have good basic science in this country, but we need to pull this through to benefit both patients and our economy," he said.

"This is a successful example of what we need to be doing. The Government accepted my report's recommendations to support this translational work and to increase the flow of successful ideas that will provide real benefit to patients."

Professor Robin Ali and James Bainbridge led the trial. A rival team of surgeons in the US carried out similar work at around the same time. Both sets of results were published recently in the New England Journal of Medicine.

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