London Doctors Cure Infant Blindness with Gene Therapy

In a groundbreaking achievement, British doctors have successfully cured blindness in four children suffering from Leber congenital amaurosis (LCA), a severe genetic condition leading to legal blindness from birth due to a defect in the AIPL1 gene. The innovative gene therapy involved injecting healthy copies of the gene into the retina through keyhole surgery, allowing the children to gain significant vision improvements, including the ability to recognize faces and read. This treatment marks the first effective intervention for the most severe form of childhood blindness and offers hope for wider applications in genetic medicine. The procedure was conducted at Moorfields Eye Hospital and the UCL Institute of Ophthalmology, with results published in The Lancet. Following the treatment, the children were monitored for five years, during which time they experienced remarkable progress in their sight. Experts are optimistic that this breakthrough could lead to earlier treatments for similar conditions in the future.