Gene therapy breakthrough for blind
Monday, 28 Apr 2008 11:52
Gene therapy treatment could be blindness breakthrough
Science In Focus
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A gene therapy breakthrough may improve the sight of thousands suffering from a congenital retinal condition.
UK researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital said the treatment for Leber's congenital amaurosis (LCA) is safe and effective in a trial of three patients.
One patient, Steven Howarth, 18, benefited from significantly improved night vision, the scientists said.
Mr Howarth could negotiate a specially constructed simulation of a night-time street scene quickly and without mistakes following surgery, whereas beforehand he could only make his way round slowly and with several mistakes.
The research team believe the operation's success was down to the fact the patient's disease had not progressed as fast as the others.
The team have already begun to trial the technique in younger patients, where they hope to achieve even better results.
Professor Robin Ali said: "Showing for the first time that gene therapy can work in patients with eye disease is a very significant milestone.
"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."
Public health minister Dawn Primarolo added: "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 one in 3,000 people."