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Visual function restoration in genetically blind mice via endogenous cellular reprogramming

By Xin Fu, Jie Zhu, Yaou Duan, Gen Li, Huimin Cai, Lianghong Zheng, Hao Qian, Changjun Zhang, Zibing Jin, Xiang-Dong Fu, Kang Zhang

Posted 08 Apr 2020
bioRxiv DOI: 10.1101/2020.04.08.030981

In this study, we developed an in-situ cellular reprogramming strategy for potent restoration of vision in advanced/end-stage retinitis pigmentosa (RP). Via repressing PTB, an RNA binding protein critical for converting non-neuronal cells to the neuronal lineage, we successfully reprogramed Muller glia to a retinal neuronal fate, and then to cones. We demonstrated that this cellular reprogramming approach was able to rescue retinal photoreceptor degeneration and restore visual functions in two RP mouse models with total blindness, suggesting a novel universal strategy for treating end-stage degenerative diseases. ### Competing Interest Statement

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