It involves young patients with a condition called Leber’s congenital amaurosis (LCA), a rare inherited eye disease caused by an abnormality in a gene called RPE65.
The condition appears at birth or in the first few months of life and causes progressive deterioration and loss of vision.
There are currently no effective treatments available.
Before the operation he completed the task slowly and made several mistakes, but following the surgery he was able to navigate quickly and without mistakes.
(Video available for media release – please see Notes to Editors.) The researchers believe the operation’s success for this particular patient could be because his disease had not progressed to the same extent as the others.
The other two patients may also still benefit from the new treatment in the future, but it will be some time before this becomes apparent.
UK researchers from the UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre have announced results from the world’s first clinical trial to test a revolutionary gene therapy treatment for a type of inherited blindness.
The results, published today in the New England Journal of Medicine, show that the experimental treatment is safe and can improve sight.
The findings are a landmark for gene therapy technology and could have a significant impact on future treatments for eye disease.The trial, conducted in the NIHR Biomedical Research Centre which received additional funding from the Department of Health, represented a world first when it began in February 2007.Crucially, the experimental treatment was found to cause no side effects in this trial.Following the treatment, the three patients involved underwent a series of tests designed to establish the effects of the therapy on vision.They all achieved levels of vision at least equivalent to before the operation, but one patient (Steven Howarth, 18) benefited from significantly improved night vision.This was demonstrated by his ability to negotiate a specially constructed simulation of a night-time street scene.