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Review

CRISPR/Cas9-Mediated Gene Correction to Understand ALS

by 1,2 and 1,2,*
1
Department of Neurosurgery, Spine and Spinal Cord Institute, College of Medicine, Yonsei University, Seoul 03722, Korea
2
Brain Korea 21 PLUS Project for Medical Science, College of Medicine, Yonsei University, Seoul 03722, Korea
*
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2020, 21(11), 3801; https://doi.org/10.3390/ijms21113801
Received: 18 April 2020 / Revised: 21 May 2020 / Accepted: 21 May 2020 / Published: 27 May 2020
(This article belongs to the Special Issue Disease Modeling Using Human Induced Pluripotent Stem Cells 2.0)
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease caused by the death of motor neurons in the spinal cord and brainstem. ALS has a diverse genetic origin; at least 20 genes have been shown to be related to ALS. Most familial and sporadic cases of ALS are caused by variants of the SOD1, C9orf72, FUS, and TARDBP genes. Genome editing using clustered regularly interspaced short palindromic repeats/CRISPR-associated system 9 (CRISPR/Cas9) can provide insights into the underlying genetics and pathophysiology of ALS. By correcting common mutations associated with ALS in animal models and patient-derived induced pluripotent stem cells (iPSCs), CRISPR/Cas9 has been used to verify the effects of ALS-associated mutations and observe phenotype differences between patient-derived and gene-corrected iPSCs. This technology has also been used to create mutations to investigate the pathophysiology of ALS. Here, we review recent studies that have used CRISPR/Cas9 to understand the genetic underpinnings of ALS. View Full-Text
Keywords: amyotrophic lateral sclerosis (ALS); CRISPR/Cas9; induced pluripotent stem cells (iPSCs); gene correction amyotrophic lateral sclerosis (ALS); CRISPR/Cas9; induced pluripotent stem cells (iPSCs); gene correction
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MDPI and ACS Style

Yun, Y.; Ha, Y. CRISPR/Cas9-Mediated Gene Correction to Understand ALS. Int. J. Mol. Sci. 2020, 21, 3801. https://doi.org/10.3390/ijms21113801

AMA Style

Yun Y, Ha Y. CRISPR/Cas9-Mediated Gene Correction to Understand ALS. International Journal of Molecular Sciences. 2020; 21(11):3801. https://doi.org/10.3390/ijms21113801

Chicago/Turabian Style

Yun, Yeomin, and Yoon Ha. 2020. "CRISPR/Cas9-Mediated Gene Correction to Understand ALS" International Journal of Molecular Sciences 21, no. 11: 3801. https://doi.org/10.3390/ijms21113801

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