Article
A mitochondrial disease model is generated and corrected using engineered base editors in rat zygotes.
Nature biotechnology - 1 Mar 2026
Chen Liang, Luan Changming, Hong Mengjia, Yuan Meng, Huang Hao, Gao Debo, Guo Xinyuan, Chen Zhengxin, Li Yongmei, Yang Lei, Yi Zongyi, Wei Wensheng, Liu Mingyao, Gao Liangcai, Han Honghui, Li Dali
Abstract excerpt
Efficient generation and correction of mutations in mitochondrial DNA (mtDNA) is challenging. Here, through embryonic injection of an mtDNA adenine base editor (eTd-mtABE), Leigh syndrome rat models were generated efficiently (up to 74%) in the F0 generation, exhibiting severe defects. To correct this mutation, a precise mtDNA C-to-T base editor was engineered and injected into mutated embryos. It achieved...
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