Article
Rs1h-/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation.
Gene therapy - 1 Aug 2022
Zeng Yong, Qian Haohua, Campos Maria Mercedes, Li Yichao, Vijayasarathy Camasamudram, Sieving Paul A
Abstract excerpt
Animal models of X-linked juvenile retinoschisis (XLRS) are valuable tools for understanding basic biochemical function of retinoschisin (RS1) protein and to investigate outcomes of preclinical efficacy and toxicity studies. In order to work with an eye larger than mouse, we generated and characterized an Rs1h-/y knockout rat model created by removing exon 3. This rat model expresses no normal RS1 protein. The...
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