Viral vectors as tools to model and treat neurodegenerative disorders.

Détails

ID Serval
serval:BIB_32D816AB1D44
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Titre
Viral vectors as tools to model and treat neurodegenerative disorders.
Périodique
Journal of Gene Medicine
Auteur(s)
Déglon N., Hantraye P.
ISSN
1099-498X (Print)
ISSN-L
1099-498X
Statut éditorial
Publié
Date de publication
2005
Volume
7
Numéro
5
Pages
530-539
Langue
anglais
Notes
Publication types: Journal Article ; Review
Résumé
The identification of disease-causing genes in familial forms of neurodegenerative disorders and the development of genetic models closely replicating human central nervous system (CNS) pathologies have drastically changed our understanding of the molecular events leading to neuronal cell death. If these achievements open new opportunities of therapeutic interventions, including gene-based therapies, the presence of the blood-brain barrier and the post-mitotic and poor regenerative nature of the target cells constitute important challenges. Efficient delivery systems taking into account the specificity of the CNS are required to administer potential therapeutic candidates. In addition, genetic models in large animals that replicate the late stages of the diseases are in most cases not available for pre-clinical studies. The present review summarizes the potential of viral vectors as tools to create new genetic models of CNS disorders in various species including primates and the recent progress toward viral gene therapy clinical trials for the administration of therapeutic candidates into the brain.
Mots-clé
Blood-Brain Barrier/drug effects, Gene Therapy, Genetic Vectors, Humans, Models, Biological, Neurodegenerative Diseases/therapy, Viruses/genetics
Pubmed
Web of science
Création de la notice
13/12/2011 17:29
Dernière modification de la notice
20/08/2019 14:18
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