Article
AAV-mediated base-editing therapy ameliorates the disease phenotypes in a mouse model of retinitis pigmentosa.
Nature communications - 15 Aug 2023
Wu Yidong, Wan Xiaoling, Zhao Dongdong, Chen Xuxu, Wang Yujie, Tang Xinxin, Li Ju, Li Siwei, Sun Xiaodong, Bi Changhao, Zhang Xueli
Abstract excerpt
Base editing technology is an ideal solution for treating pathogenic single-nucleotide variations (SNVs). No gene editing therapy has yet been approved for eye diseases, such as retinitis pigmentosa (RP). Here, we show, in the rd10 mouse model, which carries an SNV identified as an RP-causing mutation in human patients, that subretinal delivery of an optimized dual adeno-associated virus system containing the...
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