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Parents hail 'incredible' results after six-year-old girl has sight restored by gene therapy
Researchers said the one-off treatment improved vision in 15 children and may help preserve sight during critical early development.
- Six-Year-Old Saffie Sandford from Stevenage, Hertfordshire, received life-changing Luxturna gene therapy at Great Ormond Street Hospital for Leber Congenital Amaurosis . The one-off procedure, performed in April and September 2025, injects a healthy gene copy directly into each eye.
- Before treatment, Saffie struggled with low vision in daylight and total blindness in the dark, with family told she would be "blind by the age of 30" without intervention. Her mother, Lisa, noted the diagnosis at five came as a shock.
- Researchers at GOSH and University College London followed 15 children aged 15 months to 12 years, finding Luxturna strengthens visual pathways during critical development. They utilized pattern visual evoked potentials to objectively measure signal travel from retina to cortex.
- Saffie can now play on climbing frames, navigate restaurants in the evening, and safely identify hazards at school, with Lisa describing the therapy as "like someone waved a magic wand and restored her sight in the dark."
- The study's use of VEPs could establish a new standard for assessing future paediatric gene therapy trials around the world. While consultant ophthalmologist Rob Henderson noted the therapy is not a cure, researchers remain hopeful for long-term results.
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Girl, 6, has sight miraculously restored thanks to life-changing eye gene therapy on NHS
A 6-year-old girl has had her sight restored thanks to a life-changing eye gene therapy on the NHS.Saffie Sandford from Stevenage in Hertfordshire was diagnosed with a rare inherited condition called Leber's Congenital Amaurosis (LCA), which inhibits the protein creation in cells that are essential for normal vision. Children born with the condition typically have reduced vision in daylight and no vision at all in low light. The condition meant …
·London, United Kingdom
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Saffie, a 6-year-old British woman, has congenital amaurosis of Leber (ACL), a rare genetic disease that can lead to complete blindness. Thanks to revolutionary gene therapy, administered in...
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