Applications of lentiviral vectors for biology and gene therapy of neurological disorders.

Détails

ID Serval
serval:BIB_72D4069F1566
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
Applications of lentiviral vectors for biology and gene therapy of neurological disorders.
Périodique
Current Gene Therapy
Auteur(s)
Lundberg C., Björklund T., Carlsson T., Jakobsson J., Hantraye P., Déglon N., Kirik D.
ISSN
1566-5232 (Print)
ISSN-L
1566-5232
Statut éditorial
Publié
Date de publication
2008
Volume
8
Numéro
6
Pages
461-473
Langue
anglais
Notes
Publication types: Journal Article ; Review
Résumé
Recombinant lentiviral vectors (rLV) are powerful tools for gene transfer to the central nervous system (CNS) and hold great potential as a therapeutic gene therapy strategy for neurological disorders. Recent data indicate that rLVs are suitable for functional studies in the CNS by over expression or knock down of specific proteins. Based on a variety of lentiviruses species, different vector systems have been developed. However, the most commonly used rLV vector is based on the human immunodeficiency virus 1 (HIV-1). Here we describe the use of such vectors to achieve cell-specific transgene expression in the brain. In this setting, rLVs are versatile tools both due to their relatively large cloning capacity and their ability to transduce non-dividing cells. Furthermore, we discuss the preclinical development of gene therapy based on enzyme replacement and/or delivery of neurotrophic factors for neurodegenerative diseases and CNS manifestations of lysosomal storage diseases. Neuroprotective strategies that aim to deliver glial cell line-derived neurotrophic factor and ciliary neurotrophic factor for Parkinson's and Huntington's diseases in particular have been documented with success in appropriate animal models. More recently, rLVs were shown to be suitable to express small interfering RNA for treatment in models of Alzheimer's disease and amyotrophic lateral sclerosis. Finally, we present a review of the use of rLVs to model neurodegenerative diseases. rLVs have proven to be a very versatile tool to create genetic models of both Parkinson's and Huntington's diseases and thus provide possibilities to study complex genetic interactions in otherwise wild-type animals evading the necessity to create transgenic mice. Moreover, the potential of these vectors in the development of gene therapy to treat neurological disorders is considerable, which is supported by the fact that clinical trials using rLVs are underway.
Mots-clé
Animals, Ciliary Neurotrophic Factor/metabolism, Clinical Trials as Topic, Gene Expression Regulation, Gene Therapy/methods, Genetic Engineering/methods, Genetic Vectors, Glial Cell Line-Derived Neurotrophic Factor/metabolism, Humans, Huntington Disease/genetics, Huntington Disease/therapy, Lentivirus/genetics, Nerve Growth Factors/metabolism, Nervous System Diseases/genetics, Nervous System Diseases/therapy, Parkinson Disease/genetics, Parkinson Disease/therapy, RNA Interference
Pubmed
Web of science
Création de la notice
13/12/2011 17:16
Dernière modification de la notice
03/03/2018 18:18
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