Polyglutamine disorders are a complex group of incurable neurodegenerative disorders caused by an abnormal expansion in the trinucleotide cytosine-adenine-guanine tract of the affected gene. To better understand these disorders, our dependence on animal models persists, primarily relying on transgenic models. In an effort to complement and deepen our knowledge, researchers have also developed animal models of polyglutamine disorders employing viral vectors. Viral vectors have been extensively used to deliver genes to the brain, not only for therapeutic purposes but also for the development of animal models, given their remarkable flexibility. In a time- and cost-effective manner, it is possible to use different transgenes, at varying doses, in diverse targeted tissues, at different ages, and in different species, to recreate polyglutamine pathology. This paper aims to showcase the utility of viral vectors in disease modelling, share essential considerations for developing animal models with viral vectors, and provide a comprehensive review of existing viral-based animal models for polyglutamine disorders.

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多聚谷氨酰胺疾病的病毒动物模型

多聚谷氨酰胺疾病是一组复杂的无法治愈的神经退行性疾病，由受影响基因的三核苷酸胞嘧啶-腺嘌呤-鸟嘌呤束异常扩张引起。为了更好地了解这些疾病，我们仍然依赖动物模型，主要依靠转基因模型。为了补充和加深我们的知识，研究人员还利用病毒载体开发了多聚谷氨酰胺疾病的动物模型。病毒载体已被广泛用于将基因传递到大脑，不仅用于治疗目的，而且由于其卓越的灵活性也用于动物模型的开发。以时间和成本有效的方式，可以在不同的目标组织、不同的年龄和不同的物种中以不同的剂量使用不同的转基因来重建多聚谷氨酰胺病理学。本文旨在展示病毒载体在疾病建模中的实用性，分享使用病毒载体开发动物模型的基本考虑因素，并对现有的基于病毒的多谷氨酰胺疾病动物模型进行全面综述。