A mitochondrial disease model is generated and corrected using engineered base editors in rat zygotes.
basic_science · Level V
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- Record sourced from PubMed, PMID 40461781.
- Also identified by DOI 10.1038/s41587-025-02684-y.
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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 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.