Tang H, Gupte S, Xu E, Calabro KR, Friend H, Crosson SM, Fajardo D,Kostamo Z, Zhang H, Peterson JJ, Lin F, Kozmik Z, Lutz CM, Boye SL, Boye SE, Development of an AAV-Based Gene Therapy for the Ocular Phenotype of Friedreich’s Ataxia, Molecular Therapy (2025), doi:10.1016/j.ymthe.2025.10.048.
Gene supplementation via intravitreal injection of a novel AAV2-based capsid carrying FXN partially preserved retinal structure and/or function in both models, establishing proof-of-concept for this therapeutic strategy.