Department of Physiology, Korea University College of Medicine, Seoul 02841, Republic of Korea.
Department of Biomedical Sciences, Korea University College of Medicine, Seoul 02841, Republic of Korea.
Genes (Basel). 2023 Feb 14;14(2):483. doi: 10.3390/genes14020483.
Programmable nucleases, such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas, are widely accepted because of their diversity and enormous potential for targeted genomic modifications in eukaryotes and other animals. Moreover, rapid advances in genome editing tools have accelerated the ability to produce various genetically modified animal models for studying human diseases. Given the advances in gene editing tools, these animal models are gradually evolving toward mimicking human diseases through the introduction of human pathogenic mutations in their genome rather than the conventional gene knockout. In the present review, we summarize the current progress in and discuss the prospects for developing mouse models of human diseases and their therapeutic applications based on advances in the study of programmable nucleases.
可编程核酸酶，如锌指核酸酶（ZFNs）、转录激活因子样效应核酸酶（TALENs）和规律成簇间隔短回文重复序列（CRISPR）/Cas，因其多样性和在真核生物和其他动物中靶向基因组修饰的巨大潜力而被广泛接受。此外，基因组编辑工具的快速发展加速了产生各种用于研究人类疾病的基因修饰动物模型的能力。鉴于基因编辑工具的进步，这些动物模型通过在其基因组中引入人类致病突变，而不是传统的基因敲除，逐渐向模拟人类疾病的方向发展。在本综述中，我们总结了基于可编程核酸酶研究进展的人类疾病小鼠模型的开发及其治疗应用的现状和前景。
