The method presented in the article published in the prestigious journal Nature enables the ?editing? of mitochondrial DNA, meaning the introduction of precise changes within the mitochondrial genome. This method will allow further study of mitochondrial diseases caused by mutations in mitochondrial DNA and will open the way to new therapies. One of the authors of the publication is this year?s winner of the START programme of the Foundation for Polish Science (FNP), Dr. Anna Kotrys from the Institute of Biochemistry and Biophysics of the Polish Academy of Sciences in Warsaw.
In the article ?A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing,? the team to which Kotrys belongs describes a method that allows introducing changes to mitochondrial DNA for the first time. Mitochondria are ?cell?s power plants? that produce biologically useful energy (ATP). They have their own genome, separate from the nuclear one, the so-called mitochondrial DNA (mtDNA). MtDNA mutations can lead to serious diseases such as LHON (Leber’s hereditary optic neuropathy), MERRF (myoclonic epilepsy with ragged red fibers), or MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes). Moreover, mitochondrial anomalies may contribute to the development of neurodegenerative and cancerous diseases.
?The method presented in Nature enables something so far impossible: introducing precise changes within the mitochondrial genome ? its ?editing? ? meaning the direct modification of individual ?letters? of its genetic code,? explains Dr. Anna Kotrys.
The use of this new ?editor? will enable scientists to create cellular models for the study of mitochondrial diseases caused by mutations in mtDNA and will open the way to potential therapeutic applications. The development of such an mtDNA editor was possible thanks to the joint forces of three laboratories: Prof. Joseph Mougous at the University of Washington, Prof. David Liu from the Broad Institute and Harvard University, and prof. Vamsi Moothy from the Broad Institute and Massachusetts General Hospital in Boston, where Kotrys interned. There, Kotrys dealt with the last stage of the described research, namely the validation of the editor and the assessment of its operation in cells.
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In the picture: Anna Kotrys (private archive)