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

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 7...

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Bibliografische gegevens
Hoofdauteurs: Alves, Sandro, Nascimento-Ferreira, Isabel, Auregan, Gwennaëlle, Hassig, Raymonde, Dufour, Noëlle, Brouillet, Emmanuel, Pedroso de Lima, Maria C., Hantraye, Philippe, Pereira de Almeida, Luís, Déglon, Nicole
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Public Library of Science 2008
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Research Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2553199/
https://ncbi.nlm.nih.gov/pubmed/18841197
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0003341
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