Viral-based animal models in polyglutamine disorders.

Autor:	Henriques C; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., Lopes MM; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Institute for Interdisciplinary Research (III), University of Coimbra, 3030-789 Coimbra, Portugal., Silva AC; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Institute for Interdisciplinary Research (III), University of Coimbra, 3030-789 Coimbra, Portugal., Lobo DD; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Institute for Interdisciplinary Research (III), University of Coimbra, 3030-789 Coimbra, Portugal., Badin RA; CEA, DRF, Institute of Biology François Jacob, Molecular Imaging Research Center (MIRCen), 92265 Fontenay-aux-Roses, France.; CNRS, CEA, Paris-Sud University, Université Paris-Saclay, Neurodegenerative Diseases Laboratory (UMR9199), 92265 Fontenay-aux-Roses, France., Hantraye P; CEA, DRF, Institute of Biology François Jacob, Molecular Imaging Research Center (MIRCen), 92265 Fontenay-aux-Roses, France.; CNRS, CEA, Paris-Sud University, Université Paris-Saclay, Neurodegenerative Diseases Laboratory (UMR9199), 92265 Fontenay-aux-Roses, France., Pereira de Almeida L; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal., Nobre RJ; Center for Neuroscience and Cell Biology (CNC), Gene and Stem Cell Therapies for the Brain Group, University of Coimbra, 3004-504 Coimbra, Portugal.; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal.; ViraVector-Viral Vector for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Institute for Interdisciplinary Research (III), University of Coimbra, 3030-789 Coimbra, Portugal.
Jazyk:	angličtina
Zdroj:	Brain : a journal of neurology [Brain] 2024 Apr 04; Vol. 147 (4), pp. 1166-1189.
DOI:	10.1093/brain/awae012
Abstrakt:	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. (© The Author(s) 2024. Published by Oxford University Press on behalf of the Guarantors of Brain. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.)
Databáze:	MEDLINE
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