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CRISPR /Cas9 gene editing: A new therapeutic approach in the treatment of infection and autoimmunity
IUBMB Life ( IF 3.7 ) Pub Date : 2020-04-28 , DOI: 10.1002/iub.2296
Iraj Mohammadzadeh 1 , Durdi Qujeq 2, 3 , Tooba Yousefi 3 , Gordon A Ferns 4 , Mahmood Maniati 5 , Mostafa Vaghari-Tabari 6, 7
Affiliation  

CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR‐associated protein9) may be viewed as an adaptive bacterial immune system. When a virus infects a bacterium, a fragment of the virus genome is inserted into the CRISPR sequence of the bacterial genome as a memory. When the bacterium becomes infected again with the same virus, an RNA molecule that is a transcript of the memory sequence, directs Cas9, an endonuclease, to the complementary region of the virus genome, and Cas9 disables the virus by a double‐strand break. In recent years, studies have shown that by designing synthetic RNA molecules and delivering them along with Cas9 into eukaryotic cells, different regions of the cell's genome can be targeted and manipulated. These findings have drawn much attention to this new technology and it has been shown that CRISPR/Cas9 gene editing can be used to treat some human diseases. These include infectious diseases and autoimmune diseases. In this review article, in addition to a brief overview of the biology of the CRISPR/Cas9 system, we collected the most recent findings on the applications of CRISPR/Cas9 technology for better investigation of the pathogenesis and treatment of viral infections (human immunodeficiency virus infection, hepatitis virus infections, and onco‐virus infections), non‐viral infections (parasitic, fungal, and bacterial infections), and autoimmune diseases.

中文翻译:

CRISPR / Cas9 基因编辑:一种治疗感染和自身免疫的新方法

CRISPR/Cas9(成簇的规则间隔短回文重复序列/CRISPR 相关蛋白 9)可被视为一种适应性细菌免疫系统。当病毒感染细菌时,会将病毒基因组的一个片段插入到细菌基因组的 CRISPR 序列中作为记忆。当细菌再次感染同一病毒时,作为记忆序列转录本的 RNA 分子会将内切核酸酶 Cas9 引导至病毒基因组的互补区域,Cas9 通过双链断裂使病毒失效。近年来,研究表明,通过设计合成 RNA 分子并将它们与 Cas9 一起递送到真核细胞中,可以靶向和操纵细胞基因组的不同区域。这些发现引起了人们对这项新技术的广泛关注,并且已经表明CRISPR/Cas9基因编辑可用于治疗一些人类疾病。这些包括传染病和自身免疫性疾病。在这篇综述文章中,除了对 CRISPR/Cas9 系统生物学的简要概述之外,我们还收集了关于 CRISPR/Cas9 技术应用的最新发现,以更好地研究病毒感染(人类免疫缺陷病毒)的发病机制和治疗方法。感染、肝炎病毒感染和肿瘤病毒感染)、非病毒感染(寄生虫、真菌和细菌感染)和自身免疫性疾病。
更新日期:2020-04-28
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