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New Scientist has reported that the first clinical gene therapy trial to treat a vision disorder is now taking place in London, involving 12 patients with congenital conditions that cause childhood blindness. According to the report, researchers hope to restore vision in patients who have a genetic defect that causes degeneration of the retina. New Scientist says: "Robin Ali at Moorfields Eye Hospital in London and colleagues are treating adults and children with Leber's congenital amaurosis (LCA), caused by an abnormality in the RPE65 gene. This gene is important in recycling retinol, a molecule that helps the retina detect light. People with LCA usually lose vision from infancy.

"Ali's team are inserting healthy copies of RPE65 into cells in the retina, using a viral vector. Previously, dogs with LCA have had their vision restored in this way, allowing them to walk through a maze for the first time without difficulty. The team has been developing the therapy for almost 15 years, and Ali says testing it for the first time in patients represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."

According to Dr Ali: "The results from this first human trial are likely to provide an important basis for many more gene therapy protocols in the future, as well as potentially leading to an effective treatment for a rare but debilitating disease".

Although some patients in the trial have already had the procedure, the researchers say it will be many months before they know whether or not the treatment has been successful. According to Leonard Seymour, head of the Gene Delivery Group at the University of Oxford in the UK (not connected with Dr Ali's trials), the retina is a good place for gene therapy because it can be accessed by injection to overcome the problem of delivery.

"The retina is also good because it is relatively immune-privileged, meaning that the vector (in this case a virus) should not be neutralised immediately upon administration," he says.