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亨廷顿病的遗传啮齿动物模型。

Genetic Rodent Models of Huntington Disease.

机构信息

Institute of Medical Genetics and Applied Genomics, University of Tuebingen, Tuebingen, Germany.

出版信息

Adv Exp Med Biol. 2018;1049:29-57. doi: 10.1007/978-3-319-71779-1_2.

Abstract

The monogenic nature of Huntington disease (HD) has led to the development of a spectrum of useful genetically modified models. In particular, rodents have pioneered as the first HD model being generated and have since been the most widely used animal model for HD in both basic research and preclinical therapeutic studies. Based on the generation strategies, these rodent models can be classified into 3 major groups, the transgenic fragment models, the transgenic full-length models and the knock-in models. These models display a range of HD-like characteristics which resemble the clinical symptoms of HD patients. Their applications in research are thus regarded as an invaluable approach to speeding up the unraveling of the underlying pathological mechanisms of HD and for finding a disease-modifying treatment for this devastating disease. In this chapter, the similarities and differences of the most commonly used rodent HD models and their relevance to human HD will be compared and discussed. This also serves to guide the selection of an appropriate rodent HD model according to the nature of investigation.

摘要

亨廷顿病(HD)的单基因性质导致了一系列有用的基因修饰模型的发展。特别是啮齿动物开创了第一个 HD 模型的生成,并自此成为基础研究和临床前治疗研究中最广泛使用的 HD 动物模型。基于生成策略,这些啮齿动物模型可以分为 3 大组,即转基因片段模型、转基因全长模型和基因敲入模型。这些模型显示出一系列类似 HD 患者临床症状的 HD 样特征。因此,它们在研究中的应用被视为加速揭示 HD 潜在病理机制和寻找这种毁灭性疾病的疾病修饰治疗方法的宝贵途径。在本章中,将比较和讨论最常用的啮齿动物 HD 模型的异同及其与人类 HD 的相关性。这也有助于根据研究性质选择合适的啮齿动物 HD 模型。

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