First clinical trial of gene therapy for inherited blindness

The first clinical trial to test a revolutionary treatment for blindness in children has been announced by researchers at UCL (University College London).

The trial, funded by the Department of Health, is the first of its kind and could have a significant impact on future treatments for eye disease.

The trial involves adults and children who have a condition called Leber's congenital amaurosis (LCA), which is a type of inherited retinal degeneration. This disease causes progressive deterioration in vision, due to an abnormality in a particular gene called RPE65. This defect prevents normal function of the retina, the light-sensitive layer of cells at the back of the eye. This results in severely impaired vision from a very young age and there are currently no effective treatments available.

The team conducting the trial, from the UCL Institute of Ophthalmology and Moorfields Eye Hospital, is led by Professor Robin Ali and includes leading eye surgeon Mr James Bainbridge and leading retinal specialist Professor Tony Moore. The new technique involves inserting healthy copies of the gene into the cells of the retina, using a harmless virus or 'vector'. The vector has been manufactured for this trial by Targeted Genetics, of Seattle, USA.

Professor Ali said: "We have been developing gene therapy for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory. Testing it for the first time in patients is very important and exciting, and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."

To read the full press release, click on the link at the bottom of this item.

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 Links

• UCL Press release
• UCL Institute of Ophthlamology