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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2013
Materias:
Gene Regulation, Chromatin and Epigenetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3834808/
https://www.ncbi.nlm.nih.gov/pubmed/23963702
http://dx.doi.org/10.1093/nar/gkt725

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