Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.
Friedrich J, Kordasiewicz HB, O'Callaghan B, Handler HP, Wagener C, Duvick L, Swayze EE, Rainwater O, Hofstra B, Benneyworth M, Nichols-Meade T, Yang P, Chen Z, Ortiz JP, Clark HB, Öz G, Larson S, Zoghbi HY, Henzler C, Orr HT.
Friedrich J, et al. Among authors: o callaghan b, rainwater o.
JCI Insight. 2018 Nov 2;3(21):e123193. doi: 10.1172/jci.insight.123193.
JCI Insight. 2018.
PMID: 30385727
Free PMC article.