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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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| Veröffentlicht in: | PLoS One |
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| Hauptverfasser: | , , , , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Public Library of Science
2017
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| Schlagworte: | |
| Online Zugang: | 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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