𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐎𝐟𝐟𝐞𝐫𝐬 𝐋𝐢𝐟𝐞-𝐂𝐡𝐚𝐧𝐠𝐢𝐧𝐠 𝐕𝐢𝐬𝐢𝐨𝐧 𝐈𝐦𝐩𝐫𝐨𝐯𝐞𝐦𝐞𝐧𝐭𝐬 𝐟𝐨𝐫 𝐈𝐧𝐟𝐚𝐧𝐭𝐬
👶 A groundbreaking gene therapy has significantly improved vision in four infants born with severe visual impairments due to a rare genetic deficiency affecting the AIPL1 gene. This pioneering treatment, developed by researchers at University College London's Institute of Ophthalmology and Moorfields Eye Hospital, London, involves injecting healthy copies of the AIPL1 gene into the retina through keyhole surgery.
👁️ The therapy aims to preserve retinal cells and enhance their function, offering hope for children with this form of genetic blindness. Over a follow-up period of three to four years, all treated children experienced remarkable improvements in their vision, while their untreated eyes showed progressive vision loss. These findings suggest that early intervention with gene therapy can dramatically improve sight in children with severe visual impairments. The research team is now working to make this transformative therapy more widely accessible.