Review
Jul 2026
Efficacy, Safety, and Durability of Gene Therapy for Inherited Retinal Diseases in the Paediatric Population: A Scoping Review.
Paediatric benefit from IRD gene therapy depends on genotype, residual retinal structure, therapeutic platform, dose, surgical strategy, and outcome measure, and the major evidence gaps are long-term paediatric durability and inconsistent age-stratified reporting.
A. Amato, G. Iarossi, K. Stephenson
· American journal of ophthalm... · 0 citations