Peer-reviewed veterinary case report
A mitochondrial disease model is generated and corrected using engineered base editors in rat zygotes.
- Journal:
- Nature biotechnology
- Year:
- 2026
- Authors:
- Chen, Liang et al.
- Affiliation:
- Lingang Laboratory · China
- Species:
- rodent
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 Fgeneration, 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.
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Search related cases →Original publication: https://pubmed.ncbi.nlm.nih.gov/40461781/