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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5300196/
https://www.ncbi.nlm.nih.gov/pubmed/28182673
http://dx.doi.org/10.1371/journal.pone.0171127

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5300196/
https://www.ncbi.nlm.nih.gov/pubmed/28182673
http://dx.doi.org/10.1371/journal.pone.0171127

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