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CRISPR/Cas9 技术在脓毒症研究中的应用。

Application of CRISPR/Cas9 technology in sepsis research.

出版信息

Brief Funct Genomics. 2020 May 20;19(3):229-234. doi: 10.1093/bfgp/elz040.

Abstract

CRISPR/Cas9, as a new genome-editing tool, offers new approaches to understand and treat diseases, which is being rapidly applied in various areas of biomedical research including sepsis field. The type II prokaryotic CRISPR/Cas system uses a single-guide RNA (sgRNA) to target the Cas9 nuclease to a specific genomic sequence, which is introduced into disease models for functional characterization and for testing of therapeutic strategies. This incredibly precise technology can be used for therapeutic research of gene-related diseases and to program any sequence in a target cell. Most importantly, the multifunctional capacity of this technology allows simultaneous editing of several genes. In this review, we focus on the basic principles, advantages and limitations of CRISPR/Cas9 and the use of the CRISPR/Cas9 system as a powerful tool in sepsis research and as a new strategy for the treatment of sepsis.

摘要

CRISPR/Cas9 作为一种新的基因组编辑工具,为理解和治疗疾病提供了新的方法,它正在包括脓毒症领域在内的各个生物医学研究领域得到迅速应用。Ⅱ型原核 CRISPR/Cas 系统使用单指导 RNA(sgRNA)将 Cas9 核酸酶靶向特定的基因组序列,该序列被引入疾病模型中进行功能特征分析和治疗策略测试。这项令人难以置信的精确技术可用于与基因相关疾病的治疗研究,并对靶细胞中的任何序列进行编程。最重要的是,该技术的多功能性允许同时编辑几个基因。在这篇综述中,我们重点介绍了 CRISPR/Cas9 的基本原理、优势和局限性,以及将 CRISPR/Cas9 系统作为脓毒症研究的有力工具和治疗脓毒症的新策略的应用。

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