The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain

The aim of these studies was to demonstrate the therapeutic capacity of an antisense oligonucleotide with the sequence (CUG)7 targeting the expanded CAG repeat in huntingtin (HTT) mRNA in vivo in the R6/2 N-terminal fragment and Q175 knock-in Huntington’s disease (HD) mouse models. In a first study,...

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Foilsithe in:PLoS One
Main Authors: Datson, Nicole A., González-Barriga, Anchel, Kourkouta, Eleni, Weij, Rudie, van de Giessen, Jeroen, Mulders, Susan, Kontkanen, Outi, Heikkinen, Taneli, Lehtimäki, Kimmo, van Deutekom, Judith C. T.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Public Library of Science 2017
Ábhair:
Research Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5300196/
https://ncbi.nlm.nih.gov/pubmed/28182673
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0171127
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