A mitochondrial disease model is generated and corrected using engineered base editors in rat zygotes

Authors
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


Lab
China; Beijing; China; BRL Medicine; Inc.; China; China; Beijing

Journal
Nature Biotechnology

Abstract
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 restoration of wild-type alleles to an average of 53%, leading to amelioration of disease symptoms. A mitochondrial disease is modeled using engineered base editors in rats.

Keywords/Topics
Gene therapy; Molecular engineering

Source :

https://www.nature.com/articles/s41587-025-02684-y

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