科技部国家重大科学研究计划项目（课题编号：2012CBA01304）和分子发育生物学国家重点实验室经费资助。
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。
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.
杨伟莉,涂著池,李晓江. CRISPR/Cas9系统：构建非人灵长类动物疾病模型的新技术[J].中国比较医学杂志,2014,24(8):70~74.
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