Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.
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H. Zoghbi | H. Orr | G. Öz | C. Henzler | L. Duvick | H. Clark | H. Kordasiewicz | E. Swayze | M. Benneyworth | Tessa Nichols-Meade | Sarah N. Larson | Hillary P. Handler | Orion Rainwater | B. O'Callaghan | Jillian Friedrich | Judit M. Pérez Ortiz | Praseuth Yang | Bente Hofstra | C. Wagener | Zhao Chen