Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNSMichael E. Østergaard, Punit P. Seth, Eric E. Swayze et al.|Nucleic Acids Research|2013Cited by 162
In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense OligonucleotidesAmber L. Southwell, Michael R. Hayden, Niels H. Skotte et al.|Molecular Therapy|2014Cited by 148
Huntingtin suppression restores cognitive function in a mouse model of Huntington’s diseaseAmber L. Southwell, Michael R. Hayden, Holly Kordasiewicz et al.|Science Translational Medicine|2018Cited by 123
A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin allelesAmber L. Southwell, Michael R. Hayden, Niels H. Skotte et al.|Human Molecular Genetics|2017Cited by 36