Gene therapy world first hailed as major advance

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 of a revolutionary gene therapy for a type of inherited blindness.

Results from the trial, published on Monday, showed the treatment can improve vision and caused no side effects. The trial was carried out on three patients suffering from Leber's congenital amaurosis caused by an abnormality in gene RPE65. The treatment involves inserting healthy copies of the missing RPE65 gene into the cells of the retina to help them function normally. The healthy genes were delivered to the retina using a harmless virus called a vector.