A six-year-old girl from Hertfordshire has had her vision transformed after receiving pioneering gene therapy for a rare inherited form of blindness, according to multiple reports. Saffie Sandford was born with Leber's Congenital Amaurosis, a condition that severely affects sight from birth, reports said. Most children with this disorder have extremely limited vision in bright light and none in darkness, with many eventually losing sight completely as they grow older, according to reports. By the age of five, Saffie often needed a torch just to see what she was eating and struggled with balance when moving around, according to multiple reports.
In 2025, Saffie became one of the children treated with a cutting-edge gene therapy at Great Ormond Street Hospital, reports said. The procedure targeted a faulty RPE65 gene, which prevents the eye's cells from producing a protein essential for vision, according to reports. Doctors used a modified virus to deliver healthy copies of the gene directly into the back of both eyes, allowing the retinal cells to begin functioning more effectively, reports said.
We both just cried. You can't comprehend losing your eyesight - and you definitely can't comprehend it for your child.
After treatment, Saffie can recognise her parents' faces even in the dark, according to reports. She has gained confidence in movement and is now learning to ride a bike, as well as enjoying activities such as trampolining and swimming that were previously difficult or impossible for her, reports said. According to Daily Express - Health, her mother Lisa Sandford described the transformation as a miracle, saying she could not comprehend losing her child's eyesight.
Recent research from Great Ormond Street Hospital and University College London has provided further evidence supporting the therapy in young children with rare inherited blindness, according to reports. Because traditional eye tests can be unreliable at that age, researchers instead measured electrical signals travelling from the retina to the brain, reports said. Around 70% of children treated showed measurable improvements in those signals after receiving the gene therapy, according to reports.
It's like a miracle. It's like they have waved a magic wand and given Saffie her sight back. I just don't think there is a better gift than that.
The long-term durability of the treatment remains unknown, as does the total number of children treated at Great Ormond Street Hospital. Potential side effects and the cost of the therapy, including whether it is covered by healthcare systems, have not been disclosed. There are also no confirmed plans to expand this therapy to other genetic forms of blindness.