UK Girl's Vision Improves with Pioneering Gene Therapy

A six-year-old girl from Stevenage, Hertfordshire, has experienced significant improvements in her vision after receiving pioneering gene therapy for a rare inherited eye condition, allowing her to navigate daily life in ways previously impossible.

Saffie Sandford was born with Leber's Congenital Amaurosis, a disorder caused by a faulty RPE65 gene that prevents eye cells from producing a protein essential for vision. Children with the condition typically have severely limited sight in bright light and none in darkness, with many facing complete blindness in adulthood. Saffie, who had worn glasses since age two for shortsightedness, was diagnosed at five when her parents noticed her struggles in low light. Without intervention, doctors informed the family she would likely lose her sight by age 30.

In 2025, Saffie became one of the first children in the UK to receive Luxturna therapy on the National Health Service at Great Ormond Street Hospital in London. The one-time treatment involves injecting a modified virus carrying a healthy copy of the RPE65 gene directly into the back of each eye. Saffie underwent the procedure in her first eye in April and the second in September. Tests...