Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

British researchers achieve breakthrough in treating severe genetic blindness, with children experiencing life-changing improvements in vision and development.

The treatment targets Leber congenital amaurosis (LCA) caused by mutations in the AIPL1 gene, a rare condition leading to blindness within the first few years of life.
Four children underwent the gene therapy trial, which involved injecting healthy copies of the AIPL1 gene into the retina to restore photoreceptor function.
Post-treatment, children who were previously unable to perceive light can now identify shapes, recognize faces, and even read and write in some cases.
The therapy has shown sustained improvements in vision over three to four years, and additional trials with seven more children have yielded similarly promising results.
Researchers are working with regulatory agencies to make the treatment widely available within one to two years, with hopes of extending its use to other genetic blindness conditions.