Article
A conditional knockout rat resource of mitochondrial protein-coding genes via a DdCBE-induced premature stop codon.
Science advances - 14 Apr 2023
Tan Lei, Qi Xiaolong, Kong Weining, Jin Jiachuan, Lu Dan, Zhang Xu, Wang Yue, Wang Siting, Dong Wei, Shi Xudong, Chen Wei, Wang Jianying, Li Keru, Xie Yuan, Gao Lijuan, Guan Feifei, Gao Kai, Li Chaojun, Wang Cheng, Hu Zhibin, Zhang Lianfeng, Guo Xuejiang, Shen Bin, Ma Yuanwu
Abstract excerpt
Hundreds of pathogenic variants of mitochondrial DNA (mtDNA) have been reported to cause mitochondrial diseases, which still lack effective treatments. It is a huge challenge to install these mutations one by one. We repurposed the DddA-derived cytosine base editor to incorporate a premature stop codon in the mtProtein-coding genes to ablate mitochondrial proteins encoded in the mtDNA (mtProteins) instead of...
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