A six-year-old girl from Hertfordshire has had her vision transformed after receiving pioneering gene therapy, with her parents describing the change in her life as nothing short of a “miracle”. Saffie Sandford, who lives in Stevenage, was born with Leber’s Congenital Amaurosis, a rare inherited condition that severely affects sight from birth. Most children with the disorder have extremely limited vision in bright light and none in darkness, with many eventually losing sight completely as they grow older.
Daily life before treatment was extremely challenging. By the age of five, Saffie often needed a torch just to see what she was eating and struggled with balance when moving around. Her mother, Lisa Sandford, described the moment they first learned of her condition. She said: “We both just cried. You can’t comprehend losing your eyesight – and you definitely can’t comprehend it for your child.”
In 2025, Saffie became one of the children treated with a cutting-edge gene therapy at Great Ormond Street Hospital. The procedure targeted a faulty RPE65 gene, which prevents the eye’s cells from producing a protein essential for vision.
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.
The impact has been dramatic. For the first time, Saffie can recognise her parents’ faces even in the dark. She has also 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.
Lisa Sandford said the improvement feels almost unbelievable. She said: “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.”
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.
Because traditional eye tests can be unreliable at that age, researchers instead measured electrical signals travelling from the retina to the brain.
Around 70% of children treated showed measurable improvements in those signals after receiving the gene therapy.
The research team hopes these findings will lead to wider investment in similar treatments for other rare genetic eye conditions, potentially extending sight-saving benefits to more children in the future.
This story originally appeared on Express.co.uk
