In a groundbreaking breakthrough, four young infants have gained life-changing improvements in sight after receiving treatment with a pioneering genetic medicine. The UCL Institute of Ophthalmology and Moorfields Eye Hospital, in collaboration with MeiraGTx, developed the innovative therapy to target a rare genetic deficiency affecting AIPL1 gene.
Infants born with this condition suffer from severe retinal dystrophy, resulting in limited sight and eventual blindness. The treatment involves injecting healthy copies of the gene into the retina through keyhole surgery, contained within a harmless virus. This procedure has been shown to significantly improve sight in treated eyes over three to four years, but the untreated eye remains affected.
The study published in The Lancet highlights the effectiveness of early gene therapy in treating this severe form of childhood blindness. Successful gene therapy for another genetic condition has been available on the NHS since 2020. Researchers are now exploring ways to make this treatment more widely available to those with rare and common forms of genetic blindness.
Experts hail the breakthrough, citing its potential to transform lives of young children severely affected by sight impairment. The procedure was administered at Great Ormond Street Hospital, with support from the NIHR Moorfields Clinical Research Facility and Biomedical Research Center. Further research and development are underway to expand access to this groundbreaking treatment.
Source: https://medicalxpress.com/news/2025-02-genetic-therapy-infants-life-sight.html