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Girl, 6, has sight restored by life-changing gene therapy

www.bbc.com·Alex Pope·about 1 month ago
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#saffie#gene#therapy#children#family#photo
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2 hours ago Alex Pope Bedfordshire, Hertfordshire and Buckinghamshire Family handout Saffie is a massive fan of Spiderman, her family says The mother of a six-year-old girl who had life-changing eye gene therapy says it is "like someone waved a magic wand and restored her sight in the dark". Saffie Sandford, from Stevenage, has the rare inherited condition Leber's Congenital Amaurosis (LCA), which prevents cells in the eye from making a specific protein needed for normal vision. After tests were carried out at Moorfields Eye Hospital in London, she was treated at Great Ormond Street Hospital (GOSH) and underwent Luxturna therapy. Her mother Lisa said the family had been told Saffie would have been "blind by the age of 30" without treatment. Family handout Lisa, Saffie's mother, said her diagnosis had come as a huge shock GOSH said the treatment had been the first of its kind for one of the genetic causes of LCA, with Saffie having one procedure on one eye in April 2025 and the other in September.…

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