Genome editing methods in animal models
Genome editing methods in animal models
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DOI：
10.1080/19768354.2020.1726462
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发表时间：
2020-02-19
影响因子：
2.9
通讯作者：
Kim, Kyoungmi
中科院分区：
文献类型：
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作者：
Lee, Hyunji;Yoon, Da Eun;Kim, Kyoungmi
复制人类疾病的基因工程动物模型对于各种疾病的病理学研究非常重要。与使用胚胎干细胞的传统基因靶向方法相比，成簇的规则间隔短回文重复序列（CRISPR）系统的开发使动物模型的生产更快，更便宜。基于CRISPR-Cas9系统的基因组编辑工具是一项突破性技术，可以在靶DNA序列上精确引入突变。特别是，这加速了动物模型的创建，并极大地促进了利用它们的研究。在这篇综述中，我们介绍了基于CRISPR-Cas9系统用于构建人类疾病动物模型的各种策略，并描述了用于治疗目的的疾病模型的CRISPR-Cas9的各种体内递送方法。此外，我们总结了目前可用的使用CRISPR-Cas9系统生成的人类疾病动物模型，并讨论了未来的发展方向。
Genetically engineered animal models that reproduce human diseases are very important for the pathological study of various conditions. The development of the clustered regularly interspaced short palindromic repeats (CRISPR) system has enabled a faster and cheaper production of animal models compared with traditional gene-targeting methods using embryonic stem cells. Genome editing tools based on the CRISPR-Cas9 system are a breakthrough technology that allows the precise introduction of mutations at the target DNA sequences. In particular, this accelerated the creation of animal models, and greatly contributed to the research that utilized them. In this review, we introduce various strategies based on the CRISPR-Cas9 system for building animal models of human diseases and describe various in vivo delivery methods of CRISPR-Cas9 that are applied to disease models for therapeutic purposes. In addition, we summarize the currently available animal models of human diseases that were generated using the CRISPR-Cas9 system and discuss future directions.