Gene Therapy Restores Vision in Children with AIPL1-Related Retinal Dystrophy
webmaster2025-06-24T13:42:53+00:00A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...