Efficacy, Safety, and Durability of Gene Therapy for Inherited Retinal Diseases in the Paediatric Population: A Scoping Review

The authors conducted a scoping review to assess the efficacy, safety, and durability of gene therapies for inherited retinal diseases (IRDs) in children, comparing findings with adult data where possible. They identified 24 studies, with RPE65-associated disease showing the most consistent positive outcomes, while other genotypes had limited evidence of pediatric-specific efficacy. The review highlights significant gaps in long-term data and age-stratified reporting, suggesting that future trials should incorporate these elements to better understand pediatric responses to gene therapy.