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Precise modelling of mitochondrial diseases using optimized mitoBEs.

Nature - 1 Mar 2025

Zhang Xiaoxue, Zhang Xue, Ren Jiwu, Li Jiayi, Wei Xiaoxu, Yu Ying, Yi Zongyi, Wei Wensheng

Abstract excerpt

The development of animal models is crucial for studying and treating mitochondrial diseases. Here we optimized adenine and cytosine deaminases to reduce off-target effects on the transcriptome and the mitochondrial genome, improving the accuracy and efficiency of our newly developed mitochondrial base editors (mitoBEs)1. Using these upgraded mitoBEs (version 2 (v2)), we targeted 70 mouse mitochondrial DNA...

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DNA, Mitochondrial
Gene Editing
Genome, Mitochondrial
Leigh Disease
Maternal Inheritance

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Mitochondrial Diseases

Mitochondrial Proton-Translocating ATPases

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PMID: 39843744
DOI: 10.1038/s41586-024-08469-8

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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