Viral-based animal models in polyglutamine disorders

被引:0
|
作者
Henriques, Carina [1 ,2 ,3 ,4 ]
Lopes, Miguel M. [1 ,2 ,3 ,5 ]
Silva, Ana C. [1 ,2 ,3 ,5 ]
Lobo, Diana D. [1 ,2 ,3 ,5 ]
Aron Badin, Romina [6 ,7 ]
Hantraye, Philippe [6 ,7 ]
Pereira de Almeida, Luis [1 ,2 ,3 ,4 ,8 ]
Nobre, Rui Jorge [1 ,2 ,3 ,5 ]
机构
[1] Univ Coimbra, Ctr Neurosci & Cell Biol CNC, Gene & Stem Cell Therapies Brain Grp, P-3004504 Coimbra, Portugal
[2] Univ Coimbra, Ctr Innovat Biomed & Biotechnol CIBB, Vectors Gene & Cell Therapy Grp, P-3004504 Coimbra, Portugal
[3] Univ Coimbra, ViraVector Viral Vector Gene Transfer Core Facil, P-3004504 Coimbra, Portugal
[4] Univ Coimbra, Fac Pharm, P-3000548 Coimbra, Portugal
[5] Univ Coimbra, Inst Interdisciplinary Res 3, P-3030789 Coimbra, Portugal
[6] CEA, DRF, Inst Biol Francois Jacob, Mol Imaging Res Ctr MIRCen, F-92265 Fontenay Aux Roses, France
[7] Paris Sud Univ, Univ Paris Saclay, CNRS, CEA,Neurodegenerative Dis Lab UMR9199, F-92265 Fontenay Aux Roses, France
[8] Univ Coimbra, CNC Ctr Neurosci & Cell Biol, Rua Larga, P-3004504 Coimbra, Portugal
关键词
neurodegenerative disorders; brain; adeno-associated vectors; lentiviral vectors; non-human primates; rodents; MACHADO-JOSEPH-DISEASE; DOMINANT CEREBELLAR-ATAXIA; ADENOASSOCIATED VIRUS VECTOR; INCLUSION-BODY FORMATION; BULBAR MUSCULAR-ATROPHY; TRANSGENIC MOUSE MODEL; CAG REPEAT LENGTH; MUTANT HUNTINGTIN; RAT MODEL; EXPANDED POLYGLUTAMINE;
D O I
10.1093/brain/awae012
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
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. Henriques et al. 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.
引用
收藏
页码:1166 / 1189
页数:24
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