A Chinese research team reported safety and feasibility of a subretinal AAV8 gene therapy delivering a codon-optimized RS1 transgene for pediatric X-linked retinoschisis (XLRS), with results appearing in the New England Journal of Medicine. The study enrolled a dozen patients aged 5 to 18, using an AAV8 construct designed for more specific photoreceptor expression. The approach is positioned as a potential shift from supportive care to early molecular intervention if further efficacy signals emerge. The paper also notes that Chengdu Genevector helped design and manufacture the clinical-grade vectors. Atsena Therapeutics is concurrently evaluating ATSN-201, another subretinal AAV vector program for XLRS, highlighting growing clinical interest in RS1 replacement delivered directly to retinal photoreceptors.