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Doctors in London have achieved a significant milestone in the treatment of childhood blindness by successfully restoring vision in four children born with a rare genetic disorder. The groundbreaking procedure utilized pioneering gene therapy to address Leber congenital amaurosis (LCA), a severe retinal dystrophy linked to a defect in the AIPL1 gene.
The innovative treatment was carried out at Great Ormond Street Hospital, where the young patients, aged one to two and hailing from the United States, Turkey, and Tunisia, received healthy copies of the AIPL1 gene injected directly into their retinas. This procedure, performed through minimally invasive keyhole surgery, lasted approximately 60 minutes. Traditionally, children affected by LCA have very limited vision, often only able to distinguish between light and dark, with deterioration expected over time.
Following the treatment, the results were remarkable. According to findings published in The Lancet, the children exhibited significant improvements in their ability to see shapes, recognize faces, and even read and write—capabilities they would not have had without the intervention. The therapy was carefully administered to ensure safety, with only one eye treated per child, and the children will be monitored for five years to assess long-term effects.
The breakthrough has sparked hope for the future of gene therapy in treating inherited eye disorders. This treatment was developed at University College London (UCL) under a special license from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and supported by the gene therapy company MeiraGTx. Following the success of the initial four patients, additional treatments have been administered to seven more children at Evelina London Children’s Hospital, further demonstrating the potential of this advanced therapy.
As research continues, experts are optimistic that this innovative gene therapy could lead to effective treatments for other genetic eye conditions, potentially restoring sight for thousands of children worldwide. The advancement marks a significant step forward in the medical field and brings renewed hope to families affected by inherited blindness.
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