Viral-based modelling and correction of neurodegenerative diseases by RNA interference
- PMID: 16319945
- DOI: 10.1038/sj.gt.3302690
Viral-based modelling and correction of neurodegenerative diseases by RNA interference
Abstract
Experimental recapitulation of recessive human genetic neurodegenerative disease in rodents can be classically addressed through genetic disruption of the related gene. Although very informative, this specific gene targeting is restricted to mice and precludes a species scale-up towards non-human primates. Concomitantly, this requirement to silence a specific gene in a broad range of animal models is important in the design of therapeutic approaches to dominantly inherited neurodegenerative diseases. The emergence of RNA interference (RNAi), a highly specific mechanism of post-translational gene silencing, has opened a plethora of biological application ranging from reverse genetic analysis to therapeutic schemes. Recombinant viral vectors, by promoting a long-lasting delivery of genetic instructions in a broad range of cellular types of different species origins, represent potential platforms mandating silencing of specific gene products through RNAi. This review aims at providing an overview of the different viral systems engineered so far for efficient in vitro and in vivo delivery of RNAi instructions. Additionally, the potential of RNAi for functional analysis and therapy for polyglutamine disorders or amyotrophic lateral sclerosis is discussed.
Gene Therapy (2006) 13, 487-495. doi:10.1038/sj.gt.3302690; published online 1 December 2005.
Similar articles
-
Technology insight: therapeutic RNA interference--how far from the neurology clinic?Nat Clin Pract Neurol. 2007 Jul;3(7):394-404. doi: 10.1038/ncpneuro0551. Nat Clin Pract Neurol. 2007. PMID: 17611488 Review.
-
RNAi therapy for neurodegenerative diseases.Curr Top Dev Biol. 2006;75:73-92. doi: 10.1016/S0070-2153(06)75003-7. Curr Top Dev Biol. 2006. PMID: 16984810 Review.
-
Gene therapy for neurodegenerative diseases based on lentiviral vectors.Prog Brain Res. 2009;175:187-200. doi: 10.1016/S0079-6123(09)17513-1. Prog Brain Res. 2009. PMID: 19660657 Review.
-
RNA interference based gene therapy for neurological disease.Brief Funct Genomic Proteomic. 2007 Mar;6(1):40-9. doi: 10.1093/bfgp/elm005. Epub 2007 May 3. Brief Funct Genomic Proteomic. 2007. PMID: 17478451 Review.
-
Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors.Clin Sci (Lond). 2006 Jan;110(1):37-46. doi: 10.1042/CS20050158. Clin Sci (Lond). 2006. PMID: 16336203 Review.
Cited by
-
Pigment Epithelium-Derived Factor Promotes Axon Regeneration and Functional Recovery After Spinal Cord Injury.Mol Neurobiol. 2019 Nov;56(11):7490-7507. doi: 10.1007/s12035-019-1614-2. Epub 2019 May 2. Mol Neurobiol. 2019. PMID: 31049830 Free PMC article.
-
Non-viral-mediated suppression of AMIGO3 promotes disinhibited NT3-mediated regeneration of spinal cord dorsal column axons.Sci Rep. 2018 Jul 16;8(1):10707. doi: 10.1038/s41598-018-29124-z. Sci Rep. 2018. PMID: 30013050 Free PMC article.
-
Improving miRNA Delivery by Optimizing miRNA Expression Cassettes in Diverse Virus Vectors.Hum Gene Ther Methods. 2017 Aug;28(4):177-190. doi: 10.1089/hgtb.2017.036. Hum Gene Ther Methods. 2017. PMID: 28712309 Free PMC article. Review.
-
MHC universal cells survive in an allogeneic environment after incompatible transplantation.Biomed Res Int. 2013;2013:796046. doi: 10.1155/2013/796046. Epub 2013 Oct 9. Biomed Res Int. 2013. PMID: 24350288 Free PMC article.
-
An adenoviral vector-based expression and delivery system for the inhibition of wild-type adenovirus replication by artificial microRNAs.Antiviral Res. 2013 Jan;97(1):10-23. doi: 10.1016/j.antiviral.2012.10.008. Epub 2012 Nov 2. Antiviral Res. 2013. PMID: 23127366 Free PMC article.
Publication types
MeSH terms
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical
