An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says. Researchers recruited 14 people with Leber Congenital Amaurosis (LCA), a rare genetic condition that causes babies to lose some or all of their sight from birth. Eleven of the 14 had measurable improvements in the vision of one eye that received a single injection of a gene-editing medication, researchers report in the New England Journal of Medicine. “Our patients are the first congenitally blind children to be treated with gene editing, which significantly improved their daytime vision,” researcher Dr. Tomas Aleman, a pediatric ophthalmologist at the Children’s Hospital of Philadelphia, said in a news release. “While more research is needed to determine who may benefit most, we consider the early results promising,” added lead researcher Dr. Eric Pierce, director of Mass Eye and Ear Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations in Boston. “To hear from several participants how thrilled they were that they could finally see the food on their plates – that is a big deal,” Pierce said. “These were individuals who could not read any lines on an eye chart and who had no treatment options, which is the unfortunate reality for most people with inherited retinal disorders.” LCA affects 2 to 3 out… read on > read on >







