A groundbreaking gene therapy trial has shown promising results in improving vision for children born with severe childhood blindness. Conducted at Moorfields Eye Hospital in London, the experimental treatment significantly improved sight for four toddlers previously registered as legally blind. The condition, caused by a rare genetic mutation, leads to rapid vision loss from birth. Before treatment, the children could only distinguish between light and dark, but following the procedure, their vision noticeably improved.

The therapy involves injecting healthy copies of a defective gene into the eye, preserving and enhancing retinal function. Developed by scientists at University College London and administered at Great Ormond Street Hospital, the procedure was carried out under a special licence allowing experimental treatment where no alternatives exist. Unlike standard trials, this approach focused on compassionate use, ensuring children at risk of total blindness received treatment.

Assessments show all four children demonstrated better visual responses than expected. Doctors reported compelling evidence of effectiveness, with vision in treated eyes improving while untreated eyes continued to deteriorate. The treatment could improve development and social interaction for affected children.

Experts believe this marks a major advancement in gene therapy for inherited blindness. With further research, the approach could target other genetic eye conditions. Long-term monitoring will determine lasting benefits, but early results suggest life-changing potential.