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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2014
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Materias: | |
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 |