非人灵长类动物基因编辑技术的应用及挑战

doi:10.13560/j.cnki.biotech.bull.1985.2017-0891

摘要/Abstract

摘要： 建立有效的动物模型是研究人类疾病演进、开发新型治疗手段的重要方法。非人灵长类动物在进化发育、生理生化及病理方面和人类最接近,是研究人类疾病的理想动物模型。随着基因编辑技术的发展,研究者已经成功建立了多种模仿人类疾病的非人灵长类动物模型。但是CRISPR/Cas9的脱靶效应、嵌合突变以及基因敲入效率较低等突出问题也逐渐引起重视。本文综述了基因编辑技术在建立非人灵长类动物模型中的应用现状,提出了目前亟需解决的难点和应对策略,以期为高效、准确构建非人灵长类动物模型提供借鉴与参考。

关键词: 非人灵长类动物模型, TALENs, CRISPR/Cas9, 脱靶效应, 嵌合突变

Abstract: There is a pressing urgency in the medical field to establish an effective animal model to study human diseases. Non-human primates,which are evolutionary,physiologically and pathologically closer to humans than any other animal,are thus considered to be excellent animal models for studies of human diseases. With the development of gene manipulation technologies,researchers have successfully created a variety of non-human primate models via gene editing. Yet,there are still some challenges we have to face,such as off-target,mosaic mutants,and low efficiency of gene knock-in in CRISPR/Cas9. In this paper,we summarize the applications of gene editing in non-human primate models,as well as the existing problems and improvements,aimed to provide a reference for further research in non-human primate models mimicking human diseases.

Key words: non-human primate models, TALENs, CRISPR/Cas9, off-target, mosaic mutants

毕延震,肖红卫,张立苹,任红艳,华再东,华文君,王正,牛敏杰,林郑云,任习东,孙理华,郑新民. 非人灵长类动物基因编辑技术的应用及挑战[J]. 生物技术通报, 2018, 34(5): 48-56.

BI Yan-zhen, XIAO Hong-wei, ZHANG Li-ping, REN Hong-yan, HUA Zai-dong, HUA Wen-jun, WANG Zheng, NIU Min-jie, LIN Zheng-yun, REN Xi-dong, SUN Li-hua, ZHENG Xin-min. Applications and Challenges of Gene Editing in Non-human Primate Models[J]. Biotechnology Bulletin, 2018, 34(5): 48-56.

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