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CRISPR-cas 系统作为基因组工程平台:在生物医学和生物技术中的应用。

CRISPR-cas System as a Genome Engineering Platform: Applications in Biomedicine and Biotechnology.

机构信息

Department of Pharmaceutical Biotechnology, School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

出版信息

Curr Gene Ther. 2018;18(2):115-124. doi: 10.2174/1566523218666180221110627.

Abstract

Genome editing mediated by Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated proteins (Cas) has recently been considered to be used as efficient, rapid and site-specific tool in the modification of endogenous genes in biomedically important cell types and whole organisms. It has become a predictable and precise method of choice for genome engineering by specifying a 20-nt targeting sequence within its guide RNA. Firstly, this review aims to describe the biology of CRISPR system. Next, the applications of CRISPR-Cas9 in various ways, such as efficient generation of a wide variety of biomedically important cellular models as well as those of animals, modifying epigenomes, conducting genome-wide screens, gene therapy, labelling specific genomic loci in living cells, metabolic engineering of yeast and bacteria and endogenous gene expression regulation by an altered version of this system were reviewed.

摘要

CRISPR 及其相关蛋白(Cas)介导的基因组编辑最近被认为是一种高效、快速和特异性的工具,可用于修饰生物医学重要细胞类型和整个生物体中的内源性基因。通过在其向导 RNA 中指定 20 个核苷酸的靶向序列,它已成为基因组工程的一种可预测和精确的方法。首先,本综述旨在描述 CRISPR 系统的生物学。接下来,综述了 CRISPR-Cas9 在多种应用中的应用,例如高效生成各种生物医学重要的细胞模型以及动物模型、修饰表观基因组、进行全基因组筛选、基因治疗、标记活细胞中特定的基因组位点、酵母和细菌的代谢工程以及通过该系统的改变化版本调节内源性基因表达。

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