Future of human mitochondrial DNA editing technologies.

N Verechshagina, N Nikitchina, Y Yamada, Н Harashima, M Tanaka, K Orishchenko, I Mazunin

Journal: Mitochondrial DNA. Part A, DNA mapping, sequencing, and analysis 2019;30(2):214-221

PMID: 29764251

Abstract

ATP and other metabolites, which are necessary for the development, maintenance, and functioning of bodily cells are all synthesized in the mitochondria. Multiple copies of the genome, present within the mitochondria, together with its maternal inheritance, determine the clinical manifestation and spreading of mutations in mitochondrial DNA (mtDNA). The main obstacle in the way of thorough understanding of mitochondrial biology and the development of gene therapy methods for mitochondrial diseases is the absence of systems that allow to directly change mtDNA sequence. Here, we discuss existing methods of manipulating the level of mtDNA heteroplasmy, as well as the latest systems, that could be used in the future as tools for human mitochondrial genome editing.

Address: a Laboratory of Molecular Genetics Technologies , Immanuel Kant Baltic Federal University , Kaliningrad , Russia.; b Faculty of Pharmaceutical Sciences, Laboratory for Molecular Design of Pharmaceutics , Hokkaido University , Sapporo , Japan.; c Department for Health and Longevity Research , National Institutes of Biomedical Innovation, Health and Nutrition , Ibaraki City, Osaka , Japan.; d Department of Neurology , Juntendo University Graduate School of Medicine , Tokyo , Japan.; a Laboratory of Molecular Genetics Technologies , Immanuel Kant Baltic Federal University , Kaliningrad , Russia.; e Laboratory of Cell Technologies , Institute of Cytology and Genetics SB RAS , Novosibirsk , Russia.

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