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Gene treatment trial improves patient's vision

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Injecting working copies of genes into the eyes of one 18-year-old with failing sight has significantly improved his vision, London researchers have shown.

Steven Howarth is the third person to receive such experimental treatment under a team from Moorfields Eye Hospital.

Steven has Leber’s congenital amaurosis, a rare inherited eye disease caused by an abnormality in the RPE65 gene. This leads to loss of vision as individuals get older. There are currently no treatments for it.

The team, led by Professor Robin Ali, put healthy copies of the gene into retinal cells. The normal genes were transferred via a harmless virus.

All three subjects had improved vision but Steven had much better night vision – shown by testing on a simulation of a night-time street. Prior to surgery he was slow to complete the task and made mistakes. After the operation, he was quick and accurate

‘Showing for the first time that gene therapy can work in patients with eye disease is a very significant milestone,’ said Professor Ali.

‘This trial establishes proof of principle of gene therapy for inherited retinal disease and paves the way for the development of gene therapy approaches for a broad range of eye disorders.’

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