6 Year Old Saffie Has Her Vision Saved from Rare Form of Blindness Thanks to One-Time Gene Therapy

A 6-year-old girl in the UK is able to see normally again in the day and night thanks to a one-time gene therapy for a rare form of congenital blindness.

At this very tender age, Saffie Sandford from Stevenage was diagnosed with Leber’s Congenital Amaurosis, (LCA) a mutation in the RPE 65 gene which both her parents unknowingly had a copy of.

LCA typically affects children and manifests as the inability to see in low light, and the difficulty to see in daylight. Because infants and toddlers can’t sit for traditional eyesight tests, diagnosis can come late, even though its progression starts at birth. It continues worsening until blindness sets in at early adulthood.

Luckily for Saffie, Great Ormond Street Hospital (GOSH) Children’s Trust in London had been developing a novel gene therapy for this very condition by the time she arrived on referral from the Moorfields Eye Hospital in Herefordshire.

Called Luxturna, it provides a healthy copy of the RPE 65 gene directly into both eyes in a single dose. Saffie received the first one in April, 2025, and the second one in September.

“Having the treatment has been life-changing, it’s like someone waved a magic wand and restored her sight in the dark,” said Saffie’s mother, Lisa. “We’ve been able to take her trick or treating, and out to restaurants in the evening—something that was impossible before.”

“Her peripheral sight in the daylight has also improved. She’s now able to see hazards and has improved at school. She’s thriving and you wouldn’t know she had the condition just by looking at her.”

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GOSH recently published a study on the effects of Luxturna, which it developed in concert with Moorfields, administered in children as young as 15 months to as old as 12 years who had LCA. The encouraging findings showed that 7 out of 10 children showed clinically meaningful improvements in vision after receiving Luxturna.

That included personal parental anecdotes, but also results from a special new test that measures electrical signals sent to the brain from the retina—a harmless and new way of measuring vision in infants.

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“For the first time, we’ve been able to show objectively that gene therapy can strengthen the visual pathways in babies and young children who are living with this rare eye condition,” said Rob Henderson, consultant ophthalmologist at GOSH.

“For many of the families we work with, even small improvements in their child’s ability to see the world around them make a profound difference.”

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