Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles
反义寡核苷酸介导的 Ataxin-1 减少可延长 SCA1 小鼠的生存期并揭示与疾病相关的转录组谱
期刊:JCI Insight
影响因子:6.3
doi:10.1172/jci.insight.123193
Jillian Friedrich, Holly B Kordasiewicz, Brennon O'Callaghan, Hillary P Handler, Carmen Wagener, Lisa Duvick, Eric E Swayze, Orion Rainwater, Bente Hofstra, Michael Benneyworth, Tessa Nichols-Meade, Praseuth Yang, Zhao Chen, Judit Perez Ortiz, H Brent Clark, Gülin Öz, Sarah Larson, Huda Y Zoghbi, Ch