Preclinical dose-escalation study of intravitreal AAV-RS1 gene therapy in a mouse model of X-linked retinoschisis: Dose-dependent expression and improved retinal structure and function

Ronald A. Bush, Yong Zeng, Peter Colosi, Sten Kjellstrom, Suja Hiriyanna, Camasamudram Vijayasarathy, Maria Santos, Jinbo Li, Zhijian Wu, Paul A. Sieving

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Medicine & Life Sciences