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Ming SHAO, Tian-Rui XU, Ce-Shi CHEN. 2016: 基因编辑技术发展大爆炸:CRISPR/Cas9系统在疾病动物模型中的发展与应用. 动物学研究, 37(4): 191-204. DOI: 10.13918/j.issn.2095-8137.2016.4.191
引用本文: Ming SHAO, Tian-Rui XU, Ce-Shi CHEN. 2016: 基因编辑技术发展大爆炸:CRISPR/Cas9系统在疾病动物模型中的发展与应用. 动物学研究, 37(4): 191-204. DOI: 10.13918/j.issn.2095-8137.2016.4.191
Ming SHAO, Tian-Rui XU, Ce-Shi CHEN. 2016: The big bang of genome editing technology: development and application of the CRISPR/Cas9 system in disease animal models. Zoological Research, 37(4): 191-204. DOI: 10.13918/j.issn.2095-8137.2016.4.191
Citation: Ming SHAO, Tian-Rui XU, Ce-Shi CHEN. 2016: The big bang of genome editing technology: development and application of the CRISPR/Cas9 system in disease animal models. Zoological Research, 37(4): 191-204. DOI: 10.13918/j.issn.2095-8137.2016.4.191

基因编辑技术发展大爆炸:CRISPR/Cas9系统在疾病动物模型中的发展与应用

The big bang of genome editing technology: development and application of the CRISPR/Cas9 system in disease animal models

  • 摘要: 靶向基因编辑技术已经广泛用于生物医学研究。其中,与CRISPR相关的,RNA介导的内切核酸酶Cas9正成为一种强大的基因编辑工具。通过对靶基因进行高效的基因敲除,基因敲入或染色质修饰,CRISPR/Cas9系统能够有效的用于研究体内或者体外靶基因功能。该系统已经成功应用于许多生物学领域,例如遗传育种、基因功能研究以及疾病治疗。在本综述中,我们着重介绍了Cas9在疾病动物模型应用中取得的一些最新进展,挑战及对未来的方向。这种起源于细菌获得性免疫系统的新技术在不久的将来将毫无疑问地促进从基础研究到生物技术以及生物制药的飞跃发展。

     

    Abstract: Targeted genome editing technology has been widely used in biomedical studies. The CRISPR-associated RNA-guided endonuclease Cas9 has become a versatile genome editing tool. The CRISPR/Cas9 system is useful for studying gene function through efficient knock-out, knock-in or chromatin modification of the targeted gene loci in various cell types and organisms. It can be applied in a number of fields, such as genetic breeding, disease treatment and gene functional investigation. In this review, we introduce the most recent developments and applications, the challenges, and future directions of Cas9 in generating disease animal model. Derived from the CRISPR adaptive immune system of bacteria, the development trend of Cas9 will inevitably fuel the vital applications from basic research to biotechnology and bio-medicine.

     

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