Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

Huntington's disease is a fatal neurodegenerative disease caused by polyglutamine-expansion in huntingtin (HTT). Recent work showed that gene silencing approaches, including RNA interference (RNAi), improve disease readouts in mice. To advance RNAi to the clinic, we designed miHDS1, with robust...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Monteys, Alex Mas, Spengler, Ryan M., Dufour, Brett D., Wilson, Matt S., Oakley, Clayton K., Sowada, Matt J., McBride, Jodi L., Davidson, Beverly L.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
Assuntos:
RNA
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4245975/
https://ncbi.nlm.nih.gov/pubmed/25332397
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gku979
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