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World J Stem Cells. Nov 26, 2019; 11(11): 937-956
Published online Nov 26, 2019. doi: 10.4252/wjsc.v11.i11.937
CRISPR/Cas system: An emerging technology in stem cell research
Maria Teresa Valenti, Michela Serena, Luca Dalle Carbonare, Donato Zipeto
Maria Teresa Valenti, Luca Dalle Carbonare, Department of Medicine, Section of Internal Medicine D, University of Verona, Verona 37134, Italy
Michela Serena, Department of Biochemistry, University of Oxford, Oxford OX1 3QU, United Kingdom
Donato Zipeto, Department of Neurosciences, Biomedicine and Movement Sciences, Laboratory of Molecular Biology, Verona 37134, Italy
Author contributions: All authors equally contributed to this paper with conception and design of the study, literature review and analysis, drafting, critical revision and editing, and final approval of the final version.
Conflict-of-interest statement: Authors declare no conflict of interests for this article.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Corresponding author: Maria Teresa Valenti, BSc, PhD, Research Fellow, Department of Medicine, University of Verona, Ple Scuro 10, Verona 37100, Italy. mariateresa.valenti@univr.it
Telephone: +39-45-8128450 Fax: +39-45-8027403
Received: March 23, 2019
Peer-review started: March 23, 2019
First decision: August 1, 2019
Revised: August 12, 2019
Accepted: September 11, 2019
Article in press: September 11, 2019
Published online: November 26, 2019
Processing time: 228 Days and 20.5 Hours
Abstract

The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix. The ability to modify selectively specific genes provides a powerful tool for characterizing gene functions, performing gene therapy, correcting specific genetic mutations, eradicating diseases, engineering cells and organisms to achieve new and different functions and obtaining transgenic animals as models for studying specific diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 technology has recently revolutionized genome engineering. The application of this new technology to stem cell research allows disease models to be developed to explore new therapeutic tools. The possibility of translating new systems of molecular knowledge to clinical research is particularly appealing for addressing degenerative diseases. In this review, we describe several applications of CRISPR/Cas9 to stem cells related to degenerative diseases. In addition, we address the challenges and future perspectives regarding the use of CRISPR/Cas9 as an important technology in the medical sciences.

Keywords: Gene editing, CRISPR/Cas9, Stem cells, Degenerative diseases

Core tip: The possibility of translating new molecular knowledge systems to clinical research is particularly appealing for counteracting degenerative diseases as well as infective pathologies and cancer. A novel gene-editing technique, CRISPR/Cas9, has recently emerged for inducing targeted genetic modifications. Therefore, in this review, we describe recent applications of CRISPR/Cas9 to stem cells for counteracting degenerative diseases.