Overview
- Four children with leber congenital amaurosis (LCA), a rare genetic eye condition, regained significant vision after receiving gene therapy in London.
- The therapy involved injecting healthy copies of the defective AIPL1 gene into the retina, improving the function of light-sensing cells.
- The children, aged one to two at the time of treatment in 2020, showed remarkable improvements, including recognizing faces, finding toys, and even reading and writing.
- The trial was conducted by specialists at Moorfields Eye Hospital and UCL Institute of Ophthalmology, with surgeries performed at Great Ormond Street Hospital.
- Researchers aim to make the therapy more widely available, with hopes of treating both eyes and expanding its application to other genetic conditions.