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

Michael E Østergaard, Amber L Southwell, Holly Kordasiewicz, Andrew T Watt, Niels H Skotte, Crystal N Doty, Kuljeet Vaid, Erika B Villanueva, Eric E Swayze, C Frank Bennett, Michael R Hayden, Punit P Seth

105 Citations (Scopus)

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Medicine & Life Sciences