ParkinsonFranceV1, Ncbi, Curation, bibRecord, 000913

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

Identifieur interne : 000913 ( Ncbi/Curation ); précédent : 000912; suivant : 000914

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

Auteurs : Sandro Alves [Portugal, France] ; Isabel Nascimento-Ferreira [Portugal] ; Gwennaëlle Auregan [France] ; Raymonde Hassig [France] ; Noëlle Dufour [France] ; Emmanuel Brouillet [France] ; Maria C. Pedroso De Lima [Portugal] ; Philippe Hantraye [France] ; Luís Pereira De Almeida [Portugal] ; Nicole Déglon [France]

Source :

PLoS ONE [ 1932-6203 ] ; 2008.

RBID : PMC:2553199

Abstract

Recent studies have demonstrated that RNAi is a promising approach for treating autosomal dominant disorders. However, discrimination between wild-type and mutant transcripts is essential, to preserve wild-type expression and function. A single nucleotide polymorphism (SNP) is present in more than 70% of patients with Machado-Joseph disease (MJD). We investigated whether this SNP could be used to inactivate mutant ataxin-3 selectively. Lentiviral-mediated silencing of mutant human ataxin-3 was demonstrated in vitro and in a rat model of MJD in vivo. The allele-specific silencing of ataxin-3 significantly decreased the severity of the neuropathological abnormalities associated with MJD. These data demonstrate that RNAi has potential for use in MJD treatment and constitute the first proof-of-principle for allele-specific silencing in the central nervous system.

Url:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2553199

DOI: 10.1371/journal.pone.0003341
PubMed: 18841197
PubMed Central: 2553199

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PMC:2553199

Le document en format XML

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<front><div type="abstract" xml:lang="en"><p>Recent studies have demonstrated that RNAi is a promising approach for treating autosomal dominant disorders. However, discrimination between wild-type and mutant transcripts is essential, to preserve wild-type expression and function. A single nucleotide polymorphism (SNP) is present in more than 70% of patients with Machado-Joseph disease (MJD). We investigated whether this SNP could be used to inactivate mutant ataxin-3 selectively. Lentiviral-mediated silencing of mutant human ataxin-3 was demonstrated <italic>in vitro</italic>
 and in a rat model of MJD <italic>in vivo</italic>
. The allele-specific silencing of ataxin-3 significantly decreased the severity of the neuropathological abnormalities associated with MJD. These data demonstrate that RNAi has potential for use in MJD treatment and constitute the first proof-of-principle for allele-specific silencing in the central nervous system.</p>
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	La maladie de Parkinson en France (serveur d'exploration)
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La maladie de Parkinson en France (serveur d'exploration)

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

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