AGTC-RS1-001 is a first-in-human, dose-escalation Phase I/II clinical trial evaluating the safety and efficacy of intravitreal delivery of rAAV2tYF-CB-hRS1, a recombinant AAV2 vector encoding the retinoschisin (RS1) gene, for the treatment of X-linked retinoschisis (XLRS) [74]. The gene discussed is RS1; the disease is X-linked retinoschisis.