Doctors in London cure blindness in children with rare condition

In a groundbreaking achievement, doctors in London successfully cured childhood blindness in four children suffering from Leber congenital amaurosis (LCA) using advanced gene therapy. These children, legally blind from birth due to a defective AIPL1 gene, received healthy gene copies via a 60-minute keyhole surgery. Post-treatment, the children demonstrated remarkable improvements in vision, learning to see shapes, recognize faces, and even read and write. This marks a pivotal moment in gene therapy, indicating potential for early intervention in severe cases of childhood blindness, with the results published in The Lancet journal.

We have, for the first time, an effective treatment for the most severe form of childhood blindness, and a potential paradigm shift to treatment at the earliest stages of the disease.

The outcomes for these children are hugely impressive and show the power of gene therapy to change lives.

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