Article
Correction of pathogenic mitochondrial DNA in patient-derived disease models using mitochondrial base editors.
PLoS biology - 1 Jun 2025
Joore Indi P, Shehata Sawsan, Muffels Irena, Castro-Alpízar Jose, Jiménez-Curiel Elena, Nagyova Emilia, Levy Natacha, Tang Ziqin, Smit Kimberly, Vermeij Wilbert P, Rodenburg Richard, Schiffelers Raymond, Nieuwenhuis Edward E S, van Hasselt Peter M, Fuchs Sabine A, Koppens Martijn A J
Abstract excerpt
Mutations in the mitochondrial genome can cause maternally inherited diseases, cancer, and aging-related conditions. Recent technological progress now enables the creation and correction of mutations in the mitochondrial genome, but it remains relatively unknown how patients with primary mitochondrial disease can benefit from this technology. Here, we demonstrate the potential of the double-stranded DNA deaminase...
Read the complete abstract on PubMedTopics
- Humans
- DNA, Mitochondrial
- Mitochondrial Diseases
- Gene Editing
- Mitochondria