Gene therapy for a rare form of inherited blindness improves sight temporarily but can’t yet save vision cells from dying.
Light-gathering rod and cone cells continued to die in the retinas of three people despite gene therapy to correct Leber congenital amaurosis, researchers report online May 3 in the New England Journal of Medicine. A mutation in a gene called RPE65 caused the people’s blindness. That mutation impairs recycling of light-gathering proteins in the eye, leading to the death of rods and cones, also called photoreceptors.