A Chinese team reported safety and feasibility for a subretinal AAV gene therapy delivering an RS1 transgene to photoreceptors in pediatric X-linked retinoschisis. Published in the New England Journal of Medicine, the study evaluated scAAV8 vectors (using a rhodopsin kinase promoter) in a dozen patients aged 5 to 18. Investigators described the approach as more targeted to photoreceptors via promoter choice and as enabling direct delivery through subretinal administration. The report also places the work in the context of other programs, including Atsena Therapeutics’ ATSN-201, which similarly uses subretinal AAV delivery for RS1 replacement.
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