Department of Health and Social Care
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World's first gene therapy for blindness

World's first gene therapy for blindness

DEPARTMENT OF HEALTH News Release issued by The Government News Network on 28 April 2008

A Government funded trial to treat inherited blindness by administering gene therapy to the human retina has proved successful, researchers have announced today.

The trial at the Moorfields Eye Hospital/UCL Institute of Ophthalmology National Institute for Health Research Biomedical Research Centre was supported by £1 million from the Department of Health.

The trial has shown that the sight of a patient with Leber's congenital amaurosis (LCA), a type of inherited retinal degeneration which causes progressive deterioration in vision and blindness in teenagers, has made a major improvement.

This is the first major outcome from the 12 Biomedical Research Centres which were set up by the National Institute for Health Research (NIHR) in April 2007. These centres work in major areas of ill-health and clinical need to speed up the translation of fundamental science into benefits for patients. Together they will receive more than £485 million over five years.

Public Health Minister Dawn Primarolo said:

"This is a major achievement for British science and the NHS and shows we truly are at the forefront of innovation. The success of this research has huge implications for sufferers of this condition, as well as for a much larger group of inherited retinal diseases which affect 1 in 3,000 people.

"This kind of research is absolutely vital for the health and wellbeing of the nation. The Government's financial commitment to the NIHR underpins the contribution of the NHS to international scientific excellence and is helping to keep England at the top of the world biomedical research league table."

Professor Sally C. Davies, Director of Research and Development 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. I am particularly pleased that the Department of Health has contributed more than one million pounds towards this important development.

"The Moorfields Eye Hospital/UCL Institute of Ophthalmology National Institute for Health Research Biomedical Research Centre will continue to provide support, ensuring the full potential of the findings are realised and applied to treat other eye diseases. This is precisely the type of translational research that we have established our Biomedical Research Centres and Units to conduct, and will speed up the development of improved treatments for NHS patients."

Sir David Cooksey, author of the Chancellor's Cooksey Review said:

"Utilising gene therapy to prevent inherited blindness is a world first for Moorfields Eye Hospital/UCL Institute of Ophthalmology. This is a success story for the Government's Best Research for Best Health research strategy and the Biomedical Research Centres funded by the National Institute for Health Research to realise that strategy. 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.

"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 Sir John Bell, Chairman of the Office for Strategic Coordination of Health Research said:

"I am delighted about this early success in translation that has occurred within an NIHR Biomedical Research Centre. This illustrates what can happen when the capacity of the NHS is used to advance a research agenda which has direct benefits for patients."

Notes to editors

1. The National Institute for Health Research (NIHR) was set up in 2006. It is solely funded by the Department of Health to carry forward the vision and goals outlined in the Governments new health research strategy (Best Resrch for Best Health). It is provides the framework through which the research staff and research infrastructure of the NHS in England is positioned, maintained and managed as a national research facility. The NIHR provides the NHS with the support and infrastructure it needs to conduct first-class research funded by the Government and its partners alongside high-quality patient care, education and training. Its aim is to support outstanding individuals (both leaders and collaborators), working in world class facilities (both NHS and university), conducting leading edge research focused on the needs of patients

2. The National Institute for Health Research progress report "Transforming Health Research the first two years" can be downloaded from the NIHR website at:

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