A Chinese team published clinical findings in the New England Journal of Medicine supporting a subretinal gene therapy approach for X-linked retinoschisis (XLRS), using an AAV8 vector delivering a codon-optimized RS1 transgene under a rhodopsin kinase promoter. The study evaluated safety and feasibility in a small pediatric cohort (ages 5 to 18), with the authors describing retinal targeting features designed for photoreceptor expression. The work is positioned against the backdrop of limited treatment options for RS1-driven vision loss and follows the broader push toward molecularly targeted interventions in pediatric retinal dystrophies. The study’s sponsor and vector-manufacturing partners also signal the translational path for trial readiness and future efficacy studies.