London’s Moorfields Eye hospital has made history by becoming the first medical facility to successfully treat a rare genetic condition that causes blindness in children. A pioneering gene therapy treatment has given hope to four young patients born with Leber congenital amaurosis (LCA), a severe form of retinal dystrophy.
The treatment, which was developed by UCL, involves injecting healthy copies of the AIPL1 gene into the retina using keyhole surgery that took just 60 minutes. The results are “hugely impressive” and show the potential for this therapy to change lives.
Four children from the US, Turkey, and Tunisia underwent the treatment in 2020, with all showing significant improvement in their vision. One child can now read and write, while another has regained the ability to distinguish light and dark. The patients were followed up for five years, and the results were published in the Lancet journal.
The treatment was only administered into one eye per patient to minimize potential safety risks. However, some children have shown remarkable improvement, including picking tiny objects off the floor and identifying toys at a distance.
Parents of one of the treated children described their son’s transformation as “pretty amazing.” The child, who was two years old when treated, can now track light and react to stimuli that were previously impossible for him. The treatment has given new hope to families affected by this rare condition, which typically causes blindness from birth.
Source: https://www.theguardian.com/society/2025/feb/20/doctors-in-london-cure-blindness-in-children-with-rare-condition