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CRISPR 编辑在生物和生物医学研究中的应用。

CRISPR Editing in Biological and Biomedical Investigation.

机构信息

School of Psychology, Northeast Normal University, Changchun, 130021, China.

School of Basic Medicine, Changchun University of Chinese Medicine, Changchun, 130117, China.

出版信息

J Cell Biochem. 2018 Jan;119(1):52-61. doi: 10.1002/jcb.26154. Epub 2017 Jun 13.

DOI:10.1002/jcb.26154
PMID:28543738
Abstract

The CRISPR (clustered regularly interspaced short palindromic repeat)-Cas (CRISPR-associated protein) system, a prokaryotic RNA-based adaptive immune system against viral infection, is emerging as a powerful genome editing tool in broad research areas. To further improve and expand its functionality, various CRISPR delivery strategies have been tested and optimized, and key CRISPR system components such as Cas protein have been engineered with different purposes. Benefiting from more in-depth understanding and further development of CRISPR, versatile CRISPR-based platforms for genome editing have been rapidly developed to advance investigations in biology and biomedicine. In biological research area, CRISPR has been widely adopted in both fundamental and applied research fields, such as genomic and epigenomic modification, genome-wide screening, cell and animal research, agriculture transforming, livestock breeding, food manufacture, industrial biotechnology, and gene drives in disease agents control. In biomedical research area, CRISPR has also shown its extensive applicability in the establishment of animal models for genetic disorders, generation of tissue donors, implementation of antimicrobial and antiviral studies, identification and assessment of new drugs, and even treatment for clinical diseases. However, there are still several problems to consider, and the biggest concerns are the off-target effects and ethical issues of this technology. In this prospect article, after highlighting recent development of CRISPR systems, we outline different applications and current limitations of CRISPR in biological and biomedical investigation. Finally, we provide a perspective on future development and potential risks of this multifunctional technology. J. Cell. Biochem. 119: 52-61, 2018. © 2017 Wiley Periodicals, Inc.

摘要

CRISPR(成簇规律间隔短回文重复)-Cas(CRISPR 相关蛋白)系统是一种原核生物针对病毒感染的基于 RNA 的适应性免疫防御系统,正在成为广泛研究领域中强大的基因组编辑工具。为了进一步改进和扩展其功能,已经测试和优化了各种 CRISPR 传递策略,并对 Cas 蛋白等关键 CRISPR 系统组件进行了不同目的的工程改造。受益于对 CRISPR 的更深入理解和进一步发展,基于 CRISPR 的多功能平台已迅速发展,用于推进生物学和生物医学领域的研究。在生物研究领域,CRISPR 已广泛应用于基础和应用研究领域,如基因组和表观基因组修饰、全基因组筛选、细胞和动物研究、农业转化、家畜养殖、食品制造、工业生物技术以及疾病控制中的基因驱动。在生物医学研究领域,CRISPR 在遗传疾病动物模型的建立、组织供体的产生、抗菌和抗病毒研究的实施、新药的鉴定和评估、甚至临床疾病的治疗方面也显示出了广泛的适用性。然而,仍有几个问题需要考虑,而人们最关注的是这项技术的脱靶效应和伦理问题。在本文中,我们在强调 CRISPR 系统最新进展的基础上,概述了 CRISPR 在生物和生物医学研究中的不同应用和当前局限性。最后,我们对这项多功能技术的未来发展和潜在风险提供了一个展望。J. Cell. Biochem. 119:52-61, 2018. © 2017 Wiley Periodicals, Inc.

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