Moorfields trials gene therapy treatment

Moorfields Eye Hospital and the Institute of Ophthalmology have made the world's first attempt to treat blindness in children using gene therapy.

The research team conducting the first clinical trial to treat genetic sight disorders caused by the faulty gene RPE65 using gene therapy was led by Professor Robin Ali and included eye surgeon James Bainbridge and retinal specialist Professor Tony Moore.

The RPE65 defect stops the layer of cells in the retina from working and is responsible for Leber's congenital amaurosis, which is a type of inherited retinal degeneration.

The operation to correct this disorder involves injecting working copies of the defective gene into the retina. The technique has worked successfully under laboratory conditions using animals.