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CRISPR/Cas 工具在医学中的应用:全面综述。

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

机构信息

Genetic Bases of Thyroid Tumors Laboratory, Department of Morphology and Genetics, Escola Paulista de Medicina, Universidade Federal de São Paulo, São Paulo, SP, Brazil; Programa de Pós-graduação em Biociências, Universidade Federal da Integração Latino-Americana (UNILA), Foz do Iguaçu, PR, Brazil.

Reviva Research and Application Center- Lebanese University, Middle East Institute of Health University Hospital, Beirut, Lebanon.

出版信息

Gene. 2020 Jun 30;745:144636. doi: 10.1016/j.gene.2020.144636. Epub 2020 Mar 31.

DOI:10.1016/j.gene.2020.144636
PMID:32244056
Abstract

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.

摘要

自从双螺旋结构的发现和基因工程的引入以来,开发新策略来操纵基因组的可能性一直令世界各地的科学家着迷。目前,科学家们已经具备编辑基因组的知识和能力。已经建立了几种基因编辑方法,它们都像“剪刀”一样,在特定位置产生双链断裂。一种新技术的引入,该技术是从成簇规律间隔短回文重复序列(CRISPR)/Cas 细菌免疫系统中改编而来,彻底改变了基因治疗领域,因为它比以前描述的工具更能精确地编辑基因,从而可以预防和治疗人类疾病。本文旨在回顾导致 CRISPR/Cas 技术重新发现的基因组编辑历史,并探讨该引人入胜、强大、精确、简单和廉价的技术在不同领域的技术方面、应用和前景。

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