On Apr. 23, 2026, a research team at the University College London (UCL) and Great Ormond Street Hospital (GOSH) announce restoring sight to a six-year-old girl thanks to life-changing eye gene therapy for rare blindness. Saffie Sandford, from Stevenage in Hertfordshire, has the rare condition Leber’s Congenital Amaurosis (LCA), a mutation in the RPE 65 gene, that causes retinal dystrophy.

The condition prevents cells in the eye from making a specific protein that is essential for normal vision. Babies born with the condition have poor sight from infancy which further deteriorates, with many ultimately losing their vision completely in adulthood. Babies and children with the condition have low vision in daylight and no vision in low light.

Spiderman-loving Saffie was diagnosed with LCA when she was five and a half years old after her parents noticed she was struggling to see in the dark. Saffie already wore glasses and was diagnosed as short-sighted when she was two and a half.

After undergoing tests at Moorfields Eye Hospital, she was transferred to GOSH to have the eye gene therapy, Luxturna, which is the first of its kind for one of the genetic causes of LCA. Mum Lisa said: “Saffie’s diagnosis came as a huge shock to us, as we’d never heard of the condition or knew me and her dad Tam were carriers. It was such a rollercoaster of a journey, but we were so relieved and grateful when we heard there was a treatment available on the NHS for Saffie. We were told that without the treatment, she would be blind by the age of 30. “Before she had the treatment, her condition was really life-limiting. She was blind in the dark, which made daily activities challenging.”

“Her peripheral sight in the daylight has also improved. She’s now able to see hazards and has improved at school. She can also take part in normal activities that a six-year-old enjoys like playing on a climbing frame. She’s thriving and you wouldn’t know she had the condition just by looking at her.”