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Review Article

CRISPR / Cas9技术，作为在大型动物模型中概括神经退行性疾病的现代遗传操作工具

卷 21, 期 2, 2021

发表于: 14 December, 2020

页: [130 - 148] 页: 19

弟呕挨: 10.2174/1566523220666201214115024

价格: $65

摘要

背景：神经退行性疾病通常是患者中枢神经系统（CNS）结构和功能改变的结果。尽管获得了有关这些疾病的分子基础的大量基因组信息，并且由于神经系统疾病是多因素的，所以在分子水平上的病理途径与中枢神经系统疾病发展之间的因果关系仍然不清楚，需要在很大程度上加以阐明。 目的：动物模型是了解和发现病因因素在神经退行性疾病发展中的作用以及寻找适当治疗方法的便捷且有价值的工具。与啮齿动物和其他小型动物相反，大型动物，尤其是非人类的灵长类动物（NHP）与人类非常相似。因此，他们建立了合适的模型来概括啮齿动物模型中可能看不到的主要人类神经病理学表现。此外，由于它们在生理学，进化距离，解剖学和行为方面与人类相似，因此它们可作为发现神经退行性疾病的有效治疗靶点的有用模型。 方法：在这篇综述中，我们推荐基于CRISPR-Cas9系统的不同策略，用于生成人神经退行性疾病的动物模型，并解释适用于疾病模型的治疗性目的体内CRISPR-Cas9递送程序。 结果：随着CRISPR / Cas9作为基因工程领域中一种现代的特定基因编辑技术的出现，与传统的基因靶向技术相比，基因敲入和敲除等基因修饰程序变得越来越容易。与旧技术不同，这种通用技术可以有效地生成大型的转基因动物模型，而无需使实验室仪器复杂化。因此，这些动物可以准确地复制神经退行性疾病的体征。 结论：CRISPR / Cas9基因编辑技术的临床前应用为建立神经退行性疾病的动物模型提供了独特的机会，其准确性很高，并为神经系统疾病治疗和药物发现研究的突破提供了前景。此外，CRISPR在各个临床阶段的有用成果有望在短时间内转化为临床。

关键词: CRISPR / Cas9，大型动物模型，神经退行性疾病，基因组编辑，临床应用，帕金森氏病。

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