Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in Patients with X-Linked Retinoschisis: 1-Year Clinical Results.
Pennesi Mark Edward, Yang Paul, Birch David G, Weng Christina Y, Moore Anthony T, Iannaccone Alessandro et al.
This gene therapy for X-linked retinoschisis was generally safe but showed no measurable clinical benefit in vision, visual fields, or retinal structure, indicating limited efficacy for patients.