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<p>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 F<sub>0</sub> 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.</p>

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

  • 期刊论文

  • 发表日期：

    2025年6月3日

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  • Liang Chen,
  • Changming Luan,
  • Mengjia Hong,
  • Meng Yuan,
  • Hao Huang,
  • Debo Gao,
  • Xinyuan Guo,
  • Zhengxin Chen,
  • Yongmei Li,
  • Lei Yang,
  • Zongyi Yi,
  • Wensheng Wei,
  • Mingyao Liu,
  • Liangcai Gao,
  • Honghui Han,
  • Dali Li

摘要

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.