Gene therapy strengthens sight in young children born nearly blind

For children born with Leber congenital amaurosis, a severe inherited eye disease, the world can be a blur of darkness from birth. A one-time gene therapy called Luxturna (voretigene neparvovec) delivers healthy copies of the RPE65 gene directly into the eye, and on April 23, 2026, University College London reported new evidence, published in JAMA Ophthalmology, that treating children early does more than improve their eyes, it strengthens the brain's visual wiring.

Researchers at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital (GOSH) followed 15 children treated between 2020 and 2023. Using a non-invasive test called pattern visual evoked potentials, which measures how signals travel from the retina to the brain's visual cortex, the team could objectively assess even very young patients. Seven of the ten children measured showed clinically meaningful improvements, with stronger and faster visual responses. Crucially, younger children treated during a critical window of visual development gained the most. Lead researcher Dr. Rob Henderson said that for the first time they could show objectively that gene therapy can strengthen the visual pathways in babies and young children.

“A one-time gene therapy called Luxturna (voretigene neparvovec) delivers healthy copies of the RPE65 gene directly into the eye, and on April 23, 2026, University College London reported new evidence, published in JAMA Ophthalmology, that treating children early does more than improve their eyes, it strengthens the brain's visual wiring.”

The human side is vivid. Six-year-old Saffie Sandford from Hertfordshire was treated in one eye in April 2025 and the other that September. Her low-light and peripheral vision improved, she can now see hazards, she is doing better at school, and she can enjoy ordinary childhood activities like playing on a climbing frame, things that were out of reach before.

The honest caveats matter. Luxturna treats only certain RPE65-related forms of inherited blindness, the study followed a small group of children, and gene therapy remains complex and expensive. Long-term durability and broader access are open questions. Even so, objective proof that early treatment can reshape a child's developing visual brain is a profound and hopeful result. It strengthens the case for diagnosing and treating inherited blindness as young as possible, and points toward a future where more children born into darkness can grow up seeing the world.