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CRISPR/Cas9 介导的基因编辑在医学研究中的猪模型。

CRISPR/Cas9-Mediated Gene Editing in Porcine Models for Medical Research.

机构信息

Department of Pathology, West China Hospital, Sichuan University, Chendu, P.R. China.

Key Laboratory of Transplant Engineering and Immunology, Institute of Clinical Pathology, West China Hospital, Sichuan University, Chengdu, P.R. China.

出版信息

DNA Cell Biol. 2021 Dec;40(12):1462-1475. doi: 10.1089/dna.2020.6474. Epub 2021 Nov 26.

Abstract

Pigs have been extensively used as the research models for human disease pathogenesis and gene therapy. They are also the optimal source of cells, tissues, and organs for xenotransplantation due to anatomical and physiological similarities to humans. Several breakthroughs in gene-editing technologies, including the advent of clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated 9 (Cas9), have greatly improved the efficiency of genetic manipulation and significantly broadened the application of gene-edited large animal models. In this review, we have not only outlined the important applications of the CRISPR/Cas9 system in pigs as a means to study human diseases but also discussed the potential challenges of the use of CRISPR/Cas9 in large animals.

摘要

猪已被广泛用于研究人类疾病发病机制和基因治疗的模型。由于猪在解剖学和生理学上与人类相似,它们也是细胞、组织和器官异种移植的最佳来源。基因编辑技术的几项突破,包括 CRISPR/CRISPR 相关 9 (Cas9) 的出现,大大提高了遗传操作的效率,并显著拓宽了基因编辑大型动物模型的应用。在本综述中,我们不仅概述了 CRISPR/Cas9 系统在猪作为研究人类疾病的一种手段中的重要应用,还讨论了 CRISPR/Cas9 在大型动物中应用的潜在挑战。

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