Scientists in The UK successfully treated a teenage patient with a rare inherited blindness called Leber’s congenital Amaurosis (LCA). The 18 year old showed significant improved night vision after a breakthrough operation, whereas he had the ability to navigate through a simulation of a night time street with few errors. This was the worlds first landmark clinical trial to test the new gene therapy.
The trial began in early 2007 involving young patients with LCA, which is cause be a fauly gene known as RPE65 which stops the light sensitive cells in the retina from working properly. The project had two goals, first to test whether gene therapy would be safe patients with retinal disease, and secondly to test whether it could improve vision in young adults with an advanced retinal disease. In the trial, healthy copies of the faulty RPE65 into the cells of the retina of three young adults using a harmless virus to carry the gene. The vector was engineered by the US company targeted Genetics.