Article
Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.
Nucleic acids research - 1 Nov 2013
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, Bennett C Frank, Hayden Michael R, Seth Punit P
Abstract excerpt
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 oligonucleotides (ASOs) or small interfering RNAs can achieve allele selective suppression of gene expression by...
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