Retinitis pigmentosa is an inherited eye disease which affects the retina, and impacts around 1.5...
๐๐ซ๐๐๐ค๐ญ๐ก๐ซ๐จ๐ฎ๐ ๐ก ๐๐๐ง๐ ๐๐ก๐๐ซ๐๐ฉ๐ฒ ๐๐๐๐๐ซ๐ฌ ๐๐ข๐๐-๐๐ก๐๐ง๐ ๐ข๐ง๐ ๐๐ข๐ฌ๐ข๐จ๐ง ๐๐ฆ๐ฉ๐ซ๐จ๐ฏ๐๐ฆ๐๐ง๐ญ๐ฌ ๐๐จ๐ซ ๐๐ง๐๐๐ง๐ญ๐ฌ
๐ถ 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.
๐๏ธ 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.
