Medical applications of clustered regularly interspaced short palindromic repeats (CRISPR/Cas) tool: A comprehensive overview

• Published in: Gene Vol. 745; p. 144636
• Main Authors: Araldi, Rodrigo Pinheiro, Khalil, Charbel, Grignet, Pedro Henrique, Teixeira, Michelli Ramires, de Melo, Thatiana Correa, Módolo, Diego Grando, Fernandes, Luis Guilherme Virgílio, Ruiz, Jorge, de Souza, Edislane Barreiros
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier B.V 30.06.2020
• Subjects: Adaptive Immunity - genetics; Animals; Bacteria - genetics; Bacteria - immunology; Containment of Biohazards; CRISPR-Cas Systems - genetics; CRISPR-Cas Systems - immunology; CRISPR/Cas9; Disease Models, Animal; DNA Breaks, Double-Stranded; DNA Repair; Gene editing; Gene Editing - methods; Genetic Therapy - adverse effects; Genetic Therapy - methods; Humans; Personalized medicine; RNA, Guide, CRISPR-Cas Systems - genetics; Technical recommendations, medical applications; ORF; Gene editing; Technical recommendations, medical applications; HR; bp; gRNA; tracrRNA; GFP; DNMT; TIDE; DSB; TALEN; SRSR; SPY; RFP; crRNA; ssDNA; CRISPR; CD; RNAi; ZFN; CRISPR/Cas9; ZFP; dCas9; Tet1; KRAB; Personalized medicine; REC; sgRNA; PI; NHEJ; PAM; SAM
• ISSN: 0378-1119; 1879-0038
• DOI: 10.1016/j.gene.2020.144636

•Rediscovery of CRISPR/Cas system has revolutionized the gene editing field.•The variety of CRISPR/Cas systems allows many different approaches.•The CRISPR/Cas system shows a potential for both basic and translational research.•Studies are crucial to guarantee the biosafety of CRISPR/Cas for clinical purpose. Since the discovery of the double helix and the introduction of genetic engineering, the possibility to develop new strategies to manipulate the genome has fascinated scientists around the world. Currently scientists have the knowledge andabilitytoedit the genomes. Several methodologies of gene editing have been established, all of them working like “scissor”, creating double strand breaks at specific spots. The introduction of a new technology, which was adapted from the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas bacterial immune system, has revolutionized the genetic therapy field, as it allows a much more precise editing of gene than the previously described tools and, therefore, to prevent and treat disease in humans. This review aims to revisit the genome editing history that led to the rediscovery of the CRISPR/Cas technology and to explore the technical aspects, applications and perspectives of this fascinating, powerful, precise, simpler and cheaper technology in different fields.