Breakthrough in Treating Rare Genetic Blindness
Four children with a severe form of childhood blindness due to AIPL1 gene deficiency underwent successful gene therapy, showing significant sight improvement. The treatment involved injecting healthy gene copies into the retina.
The therapy, developed by UCL and , represents a potential paradigm shift in treating early-stage genetic blindness, with remarkable outcomes such as children being able to distinguish shapes and recognize faces.
Global Impact and Future Prospects
The treatment, initially trialed on children from the , , and , has opened new avenues for addressing both rare and common forms of genetic blindness worldwide.
Researchers are now focused on making this groundbreaking therapy more widely available, with ongoing studies to confirm and expand on the early successes reported in .