Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

Autosomal dominant diseases such as Huntington’s disease (HD) are caused by a gain of function mutant protein and/or RNA. An ideal treatment for these diseases is to selectively suppress expression of the mutant allele while preserving expression of the wild-type variant. RNase H active antisense ol...

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Autors principals: Østergaard, Michael E., Southwell, Amber L., Kordasiewicz, Holly, Watt, Andrew T., Skotte, Niels H., Doty, Crystal N., Vaid, Kuljeet, Villanueva, Erika B., Swayze, Eric E., Frank Bennett, C., Hayden, Michael R., Seth, Punit P.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2013
Matèries:
Gene Regulation, Chromatin and Epigenetics
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3834808/
https://ncbi.nlm.nih.gov/pubmed/23963702
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkt725
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