The parents of a six-year-old girl whose eyesight has been restored by gene therapy have described the results as a “miracle”.
Saffie Sandford, from Stevenage in Hertfordshire, has a rare genetic condition called Leber’s Congenital Amaurosis, which causes blindness.
Children with the condition typically have very little vision in daylight, and none in low light. It can lead to complete loss of sight by adulthood.
By the age of five, Saffie needed to use a torch to eat her meals and was unsteady on her feet.
Saffie’s mum, Lisa Sandford, spoke to ITV News about the day they received the diagnosis.
“We both just cried,” said Lisa. “You can’t comprehend losing your eyesight - and you definitely can’t comprehend it for your child.”
But in 2025, Saffie had life-changing gene therapy at Great Ormond Street Hospital (GOSH).
Saffie has a faulty RPE 65 gene, which prevents cells in the eye making a protein needed for vision.
A modified virus was injected directly into the back of Saffie’s eyes, introducing healthy copies of the gene into the cells.
Saffie can now, for the first time, see her parents’ faces in the dark; she has learnt to ride a bike, enjoys trampolining and swimming – all activities that she struggled to do before the treatment.
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