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https://hdl.handle.net/10316/12776
Título: | Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease | Autor: | Alves, Sandro Nascimento-Ferreira, Isabel Auregan, Gwennaëlle Hassig, Raymonde Dufour, Noëlle Brouillet, Emmanuel Lima, Maria C. Pedroso de Hantraye, Philippe Almeida, Luís Pereira de Déglon, Nicole |
Data: | Out-2008 | Editora: | Public Library of Science | Citação: | PloS One. 3:10 (2008) e3341 | Resumo: | 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 | URI: | https://hdl.handle.net/10316/12776 | ISSN: | 1932-6203 | DOI: | 10.1371/journal.pone.0003341 | Direitos: | openAccess |
Aparece nas coleções: | FFUC- Artigos em Revistas Internacionais FCTUC Ciências da Vida - Artigos em Revistas Internacionais |
Ficheiros deste registo:
Ficheiro | Descrição | Tamanho | Formato | |
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Allele-Specific RNA Silencing of Mutant Ataxin-3.pdf | 1.25 MB | Adobe PDF | Ver/Abrir |
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