The prospects of gene therapy for mitochondrial diseases: can’t we do without CRISPR/Cas9?

Chicherin IV1, Levitskii SA1, Krasheninnikov IA1, Tarassov I2, Kamenski P1,3
About authors

1 Faculty of Biology,
Lomonosov Moscow State University, Moscow, Russia

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

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