聚谷氨酰胺束
动物模型
病毒载体
生物
转基因
计算生物学
神经科学
疾病
病毒学
生物信息学
基因
医学
遗传学
亨廷顿蛋白
病理
内分泌学
重组DNA
突变体
作者
Carina Henriques,Miguel M Lopes,Ana C Silva,Diana Lobo,Romina Aron Badin,Philippe Hantraye,Luís Pereira de Almeida,Rui Jorge Nobre
出处
期刊:Brain
[Oxford University Press]
日期:2024-01-29
卷期号:147 (4): 1166-1189
标识
DOI:10.1093/brain/awae012
摘要
Abstract 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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