00:03Six-year-old Safie Sandford, who is from Stevenage, has the rare inherited condition
00:08Leber's congenital amaurosis, also known as LCA, which prevents cells in the eye from
00:15making a specific protein needed for normal vision. Babies and children with the condition
00:20have low vision in daylight and no vision in low light, and can lose their sight completely
00:27in adulthood. She was diagnosed with LCA when she was five after her parents noticed she
00:33was struggling to see in the dark. Moorfields Eye Hospital in London carried out tests to
00:40diagnose Safie, and then she was transferred to Great Ormond Street Hospital to have the
00:45eye gene therapy called Lux-Turner. This treatment is the first of its kind for one of the genetic
00:53causes of LCA. The one-off therapy contains a healthy copy of the gene, which is injected
00:59directly into the eye. Safie's mother said Safie's diagnosis came as a huge shock to the family,
01:06as they'd never heard of the condition or knew that her or her daughter's father, Tam, were
01:12carriers. Safie underwent eye gene therapy in her first eye in April 2025, and then in her
01:19second eye in September. She already wore glasses, having been diagnosed as short-sighted when
01:24she was two.
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