ISSN Print 2500–1094
ISSN Online 2542–1204
BIOMEDICAL JOURNAL OF PIROGOV UNIVERSITY (MOSCOW, RUSSIA)
Copyright: © 2017 by the authors. Licensee: Pirogov University.
This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution license (CC BY).
This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution license (CC BY).
OPINION
The prospects of gene therapy for mitochondrial diseases: can’t we do without CRISPR/Cas9?
About authors
1 Faculty of Biology, Lomonosov Moscow State University, Moscow
2 Laboratory of Molecular Genetics, Genomics and Microbiology,University of Strasbourg, Strasbourg, France
3 Institute of Living Systems,Immanuel Kant Baltic Federal University, Kaliningrad, Russia
Correspondence should be addressed: Peter Kamenski
Leninskie Gory, d. 1, str. 12, Moscow, Russia, 119991 (Faculty of Biology); ur.usm.oib.nietorp@retep
About paper
Funding: Russian Science Foundation, grant No. 14-50-00029 (Lomonosov Moscow State University); Project 5–100 of the Ministry of Education and Science of the Russian Federation (Immanuel Kant Baltic Federal University); the International Associated Laboratory RNA-mitocure (Lomonosov Moscow State University and the University of Strasbourg).
Contribution of the authors to this work: Chicherin IV, Levitsky SA —analysis of literature; Krasheninnikov IA, Tarassov I — analysis of mechanism of a hypothetical mitoCRISPR/Cas9 platform; Kamenski P — data generalization, drafting of a manuscript. All authors participated in editing of the manuscript.
Received: 2017-06-20
Accepted: 2017-06-23
Published online: 2017-07-19
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Mitochondrial DNA mutations cause severe inherited disorders in humans. To date, there are a few therapeutic strategies for their correction; however, it is highly unlikely that they would be routinely used in clinical practice. The past few years have witnessed the rapid progress of a genome editing technology known as CRISPR/Cas9. The present review focuses on the current strategies to combat mitochondrial mutations and reveals their major drawbacks. The article also explores the possibility of creating a possible specific CRISPR/Cas9 tool for correcting mitochondrial DNA mutations and provides a rough description of its mechanism of action. A particular focus is paid to technical challenges. On the whole, we see no principal barriers to implementing a mitoCRISPR/Cas9 system for treating mitochondrial disorders.
Keywords: genome editing, gene therapy, CRISPR/Cas9, mitochondrial DNA, mitochondrial diseases