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Int. J. Mol. Sci. 2018, 19(3), 906; https://doi.org/10.3390/ijms19030906

CRISPR/Cas9 Technology as an Emerging Tool for Targeting Amyotrophic Lateral Sclerosis (ALS)

1
Department of Pathophysiology, School of Medicine, Collegium Medicum, University of Warmia and Mazury, 10-900 Olsztyn, Poland
2
Department of Neurology and Neurosurgery, School of Medicine, Collegium Medicum, University of Warmia and Mazury, 10-900 Olsztyn, Poland
3
Laboratory for Regenerative Medicine, School of Medicine, Collegium Medicum, University of Warmia and Mazury, 10-900 Olsztyn, Poland
*
Authors to whom correspondence should be addressed.
Received: 13 February 2018 / Revised: 13 March 2018 / Accepted: 16 March 2018 / Published: 19 March 2018
(This article belongs to the Special Issue Rare Diseases: Molecular Mechanisms and Therapeutic Strategies)
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Abstract

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) is a genome editing tool that has recently caught enormous attention due to its novelty, feasibility, and affordability. This system naturally functions as a defense mechanism in bacteria and has been repurposed as an RNA-guided DNA editing tool. Unlike zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), CRISPR/Cas9 takes advantage of an RNA-guided DNA endonuclease enzyme, Cas9, which is able to generate double-strand breaks (DSBs) at specific genomic locations. It triggers cellular endogenous DNA repair pathways, contributing to the generation of desired modifications in the genome. The ability of the system to precisely disrupt DNA sequences has opened up new avenues in our understanding of amyotrophic lateral sclerosis (ALS) pathogenesis and the development of new therapeutic approaches. In this review, we discuss the current knowledge of the principles and limitations of the CRISPR/Cas9 system, as well as strategies to improve these limitations. Furthermore, we summarize novel approaches of engaging the CRISPR/Cas9 system in establishing an adequate model of neurodegenerative disease and in the treatment of SOD1-linked forms of ALS. We also highlight possible applications of this system in the therapy of ALS, both the inherited type as well as ALS of sporadic origin. View Full-Text
Keywords: CRISPR/Cas9; ALS; ALS models; ALS therapy CRISPR/Cas9; ALS; ALS models; ALS therapy
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Kruminis-Kaszkiel, E.; Juranek, J.; Maksymowicz, W.; Wojtkiewicz, J. CRISPR/Cas9 Technology as an Emerging Tool for Targeting Amyotrophic Lateral Sclerosis (ALS). Int. J. Mol. Sci. 2018, 19, 906.

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