利用 CRISPR/Cas9 技术建立脑部疾病模型。
Use of CRISPR/Cas9 to model brain diseases.
机构信息
Ministry of Education CNS Regeneration Collaborative Joint Laboratory, Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou 510631, China.
Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, USA.
出版信息
Prog Neuropsychopharmacol Biol Psychiatry. 2018 Feb 2;81:488-492. doi: 10.1016/j.pnpbp.2017.04.003. Epub 2017 Apr 6.
Aging-related brain diseases consist of a number of important neurodegenerative disorders, including Alzheimer's, Parkinson's, and Huntington's diseases, all of which have become more prevalent as the life expectancy of humans is prolonged. Age-dependent brain disorders are associated with both environmental insults and genetic mutations. For those brain disorders that are inherited, gene editing is an important tool for establishing animal models to investigate the pathogenesis of disease and identify effective treatments. Here we focus on the tools for gene editing, especially CRISPR/Cas9, and discuss their application for generating animal models that can recapitulate the brain pathology seen in human diseases. We also highlight the advantages and disadvantages of establishing genetically modified animal models. Finally, we discuss recent findings to resolve technical issues related to the use of CRISPR/Cas9 for generating animal models of brain diseases.
与年龄相关的脑部疾病包括多种重要的神经退行性疾病，如阿尔茨海默病、帕金森病和亨廷顿病等，随着人类预期寿命的延长，这些疾病的发病率都有所上升。与年龄相关的脑部疾病既与环境损伤有关，也与基因突变有关。对于那些遗传性脑部疾病，基因编辑是建立动物模型以研究疾病发病机制和确定有效治疗方法的重要工具。在这里，我们重点介绍基因编辑工具，特别是 CRISPR/Cas9，并讨论它们在生成可重现人类疾病脑部病理学的动物模型中的应用。我们还强调了建立基因修饰动物模型的优缺点。最后，我们讨论了最近的研究结果，以解决与使用 CRISPR/Cas9 生成脑部疾病动物模型相关的技术问题。
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