Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review)

International Journal of Molecular Medicine
Diana Raquel Rodríguez-RodríguezHugo Alberto Barrera-Saldaña

Abstract

Genome editing reemerged in 2012 with the development of CRISPR/Cas9 technology, which is a genetic manipulation tool derived from the defense system of certain bacteria against viruses and plasmids. This method is easy to apply and has been used in a wide variety of experimental models, including cell lines, laboratory animals, plants, and even in human clinical trials. The CRISPR/Cas9 system consists of directing the Cas9 nuclease to create a site‑directed double‑strand DNA break using a small RNA molecule as a guide. A process that allows a permanent modification of the genomic target sequence can repair the damage caused to DNA. In the present study, the basic principles of the CRISPR/Cas9 system are reviewed, as well as the strategies and modifications of the enzyme Cas9 to eliminate the off‑target cuts, and the different applications of CRISPR/Cas9 as a system for visualization and gene expression activation or suppression. In addition, the review emphasizes on the potential application of this system in the treatment of different diseases, such as pulmonary, gastrointestinal, hematologic, immune system, viral, autoimmune and inflammatory diseases, and cancer.

Citations

Feb 11, 2020·Clinical Genetics·Wei ZhangHongbo Wang
Jun 28, 2019·MSphere·Benedikt M MarkusSebastian Lourido
Jul 11, 2020·Journal of Human Hypertension·Durairaj SekarBiruntha M
Oct 28, 2020·Cells·Daniel J TurnhamHelen B Pearson
Feb 27, 2021·Frontiers in Cellular Neuroscience·Stefanie PerrierGeneviève Bernard
Jun 3, 2021·Viruses·Nathália Alves Araújo de AlmeidaVanessa Salete de Paula
Mar 25, 2021·American Journal of Physiology. Gastrointestinal and Liver Physiology·Sabrina T HirshornYana Zavros

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Methods Mentioned

BETA
transfection
exome sequencing
gene knockout
genetic modification
transgenic
xenograft

Software Mentioned

- Cas
sgRNA Designer
CRISP MultiTarget
CHOPCHOP
CCtop
CRISPR
CRISPRdirect
CRISPOR
Split
Breaking

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