'It's like someone waved a magic wand': Girl, 6, has sight restored by gene therapy
Summary
A six-year-old girl named Saffie Sandford, who has a rare inherited eye condition called Leber's Congenital Amaurosis (LCA), received a gene therapy that improved her vision. The treatment involved injecting a healthy gene into each eye, helping her see better in both low and bright light.Key Facts
- Saffie Sandford is six years old and from Stevenage.
- She has LCA, a rare genetic eye condition that causes poor vision and can lead to blindness by adulthood.
- Doctors treated her with Luxturna, a gene therapy that injects a healthy gene into the eye.
- She had two procedures, one on each eye, months apart in 2025 at Great Ormond Street Hospital.
- The treatment improved her ability to see in the dark and her peripheral (side) vision during the day.
- Saffie was diagnosed at age five after noticing trouble seeing in low light; she had worn glasses since age two.
- Research shows gene therapy works best in very young children during critical brain development periods.
- The therapy is not a cure but can strengthen visual pathways and improve sight for children with inherited retinal conditions.
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