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CRISPR-Cas9 技术用于创建能够模拟和治疗疾病的生物替身:从发现到最新进展。

CRISPR-Cas9 Technology for the Creation of Biological Avatars Capable of Modeling and Treating Pathologies: From Discovery to the Latest Improvements.

机构信息

Univ. Grenoble Alpes, CEA, INSERM, 38000 Grenoble, France.

出版信息

Cells. 2022 Nov 15;11(22):3615. doi: 10.3390/cells11223615.

Abstract

This is a spectacular moment for genetics to evolve in genome editing, which encompasses the precise alteration of the cellular DNA sequences within various species. One of the most fascinating genome-editing technologies currently available is Clustered Regularly Interspaced Palindromic Repeats (CRISPR) and its associated protein 9 (CRISPR-Cas9), which have integrated deeply into the research field within a short period due to its effectiveness. It became a standard tool utilized in a broad spectrum of biological and therapeutic applications. Furthermore, reliable disease models are required to improve the quality of healthcare. CRISPR-Cas9 has the potential to diversify our knowledge in genetics by generating cellular models, which can mimic various human diseases to better understand the disease consequences and develop new treatments. Precision in genome editing offered by CRISPR-Cas9 is now paving the way for gene therapy to expand in clinical trials to treat several genetic diseases in a wide range of species. This review article will discuss genome-editing tools: CRISPR-Cas9, Zinc Finger Nucleases (ZFNs), and Transcription Activator-Like Effector Nucleases (TALENs). It will also encompass the importance of CRISPR-Cas9 technology in generating cellular disease models for novel therapeutics, its applications in gene therapy, and challenges with novel strategies to enhance its specificity.

摘要

这是遗传学在基因组编辑领域发展的一个非凡时刻,涵盖了对各种物种细胞 DNA 序列的精确改变。目前最引人入胜的基因组编辑技术之一是成簇规律间隔短回文重复序列 (CRISPR) 及其相关蛋白 9 (CRISPR-Cas9),由于其有效性,它在短时间内深入到研究领域。它已成为广泛应用于生物学和治疗学应用的标准工具。此外,还需要可靠的疾病模型来提高医疗质量。CRISPR-Cas9 有可能通过生成细胞模型来丰富我们对遗传学的认识,这些模型可以模拟各种人类疾病,从而更好地了解疾病后果并开发新的治疗方法。CRISPR-Cas9 提供的基因组编辑精度现在为基因治疗在临床试验中的扩展铺平了道路,以治疗多种遗传疾病和广泛的物种。本文将讨论基因组编辑工具:CRISPR-Cas9、锌指核酸酶 (ZFNs) 和转录激活因子样效应核酸酶 (TALENs)。它还将涵盖 CRISPR-Cas9 技术在生成用于新型治疗的细胞疾病模型中的重要性、它在基因治疗中的应用以及增强其特异性的新策略所面临的挑战。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5b55/9688409/89ebf9096cfd/cells-11-03615-g001.jpg

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