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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2014
Materias:	RNA
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4245975/ https://www.ncbi.nlm.nih.gov/pubmed/25332397 http://dx.doi.org/10.1093/nar/gku979

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