Stevenage schoolgirl has sight restored after landmark NHS eye gene therapy at GOSH
WHO: Great Ormond Street Hospital (GOSH) and Rob Henderson
WHAT: Restoration of sight using Luxturna gene therapy for LCA
WHERE: Great Ormond Street Hospital, London
WHEN: Results published 22 April 2026
WHY: To improve visual pathways and prevent total blindness in children with RPE65 mutations
A Stevenage schoolgirl has had her vision saved by NHS eye gene therapy after facing total blindness by age 30.
Life-changing results for NHS eye gene therapy
Six-year-old Saffie Sandford is thriving after receiving a first-of-its-kind treatment for Leber’s Congenital Amaurosis (LCA). This rare condition is caused by a mutation in the RPE65 gene. It prevents the eye from producing a protein essential for vision.
Saffie underwent the one-off therapy in her first eye in April 2025. Clinicians treated her second eye in September 2025. Her mother, Lisa, described the impact as restorative.
Lisa said: “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.”
Strengthening visual brain pathways
Researchers at GOSH and University College London (UCL) followed 15 children treated between 2020 and 2023. This represents the largest UK study of its kind. The study utilized non-invasive testing to measure signals from the retina to the brain.
Data shows that 7 out of 10 children who completed testing showed clinically meaningful improvements. These included stronger visual signals and faster processing times. Clinicians treated patients ranging from 15 months to 12 years old.
Consultant ophthalmologist Rob Henderson led the research. He noted the importance of treating children during critical stages of brain development.
Rob Henderson said: “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.”
He added: “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.”
Great Ormond Street Hospital is a world-renowned centre of excellence. It has been at the forefront of developing gene therapies for 25 years. The hospital launched this specific joint service with Moorfields Eye Hospital in 2020.
Saffie's peripheral vision in daylight has also improved. This has allowed her to navigate hazards more safely and improve her performance at school. Her family remains grateful for the opportunity provided by the NHS.
OFFICIAL SOURCE VERIFICATION:
This report is based on official clinical data from NHS Trust / Health Authority.
Document: NHS eye gene therapy restores Saffie's sight
Source Link: https://www.gosh.nhs.uk/news/nhs-eye-gene-therapy-restores-saffies-sight/
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