CAJ | 학술논문

动物疾病模型在研究人类疾病致病机理和药物筛选中起到了关键作用。非人灵长类动物由于与人类更为接近，在探究人类神经退行性疾病、神经精神疾病及人类认知功能、神经环路等方面具有巨大的优势可成为研究和药物筛选的重要疾病模型。然而，由于缺乏大动物的胚胎干细胞系，传统的基因打靶技术难于用来建立灵长类动物疾病模型。最近发展的基因编辑新技术 CRISPR /Cas9系统在定向对基因进行修饰上展现出了巨大的潜力。本文将介绍 CRISPR /Cas9技术的发展和应用，以及非灵长类动物作为神经退行性疾病模型的优势和意义。
동물질병모형재연구인류질병치병궤리화약물사선중기도료관건작용。비인령장류동물유우여인류경위접근，재탐구인류신경퇴행성질병、신경정신질병급인류인지공능、신경배로등방면구유거대적우세가성위연구화약물사선적중요질병모형。연이，유우결핍대동물적배태간세포계，전통적기인타파기술난우용래건립령장류동물질병모형。최근발전적기인편집신기술 CRISPR /Cas9계통재정향대기인진행수식상전현출료거대적잠력。본문장개소 CRISPR /Cas9기술적발전화응용，이급비령장류동물작위신경퇴행성질병모형적우세화의의。
Animal models are highly valuable systems that have been extensively used to elucidate human disease pathogenesis and to find therapeutic ways to treat human diseases .Since non-human primates are close to humans,monkeys are important model species in exploring the mechanisms and treatment of human neurodegenerative diseases , neuropsychiatric disorders, cognitive function, and neural circuits.However, due to the lack of embryonic stem cell lines in large animals, the traditional gene targeting technology is difficult to establish primate animal models of human diseases . CRISPR/Cas9, as a recently developed tool for genome modifications , has been successfully used to target genomic loci in mouse, rat, monkey, and other species.Here, we discuss the utilization of CRISPR /Cas9 technology in establishing monkey models for studying human neurodegenerative diseases .