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World's first gene therapy for blindness

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.

• 28 April 2008 - news story (opens new window)