“A six-year-old girl has had her sight restored thanks to life-changing eye gene therapy for rare blindness at Great Ormond Street Hospital (GOSH).
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…
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…
Saffie had the eye gene therapy in her first eye in April 2025 just before her sixth birthday, and in her second eye in September 2025.
‘Having the treatment has been life-changing, it’s like someone waved a magic wand and restored her sight in the dark. We’ve been able to take her trick or treating, and out to restaurants in the evening – something that was impossible before.'”
