Strand-selective base editing of human mitochondrial DNA using mitoBEs
Mitochondrial DNA (mtDNA) mutations are associated with many human diseases, and around 95% of these are point mutations that could potentially be corrected using base editing approaches. Therefore, there is a high demand for technologies that enable mtDNA base editing, which could aid in understanding the underlying mechanisms of pathogenesis and developing cures for these diseases. Although the CRISPR system has been widely used for nuclear genome base editing. it is currently impractical to apply this system for editing the mitochondrial genome due to the absence of an effective method for delivering guide RNA into this organelle.
mitoBEs offer a precise, efficient DNA editing tool with broad applicability for therapy in mitochondrial genetic diseases.
By combining TALE–nickase and TALE–deaminase, we develop mitochondrial base editing tools, named mitoBEs.
Mitochondrial base editing techniques are relatively new editing tools that could make specific base substitutions of mtDNA without causing the double-strand breaks that could cause rapid degradation of mtDNA.
The realization of targeted mitochondrial base substitutions could greatly empower researchers to study the effects of specific mtDNA mutations and correct disease-causing point mutations for therapeutic purposes.
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