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Decreasing mutant ATXN1 nuclear localization improves a spectrum of SCA1-like phenotypes and brain region transcriptomic profiles.
Handler HP, Duvick L, Mitchell JS, Cvetanovic M, Reighard M, Soles A, Mather KB, Rainwater O, Serres S, Nichols-Meade T, Coffin SL, You Y, Ruis BL, O'Callaghan B, Henzler C, Zoghbi HY, Orr HT. Handler HP, et al. Among authors: rainwater o. Neuron. 2023 Feb 15;111(4):493-507.e6. doi: 10.1016/j.neuron.2022.11.017. Epub 2022 Dec 27. Neuron. 2023. PMID: 36577403 Free PMC article.
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.
Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment.
O'Callaghan B, Hofstra B, Handler HP, Kordasiewicz HB, Cole T, Duvick L, Friedrich J, Rainwater O, Yang P, Benneyworth M, Nichols-Meade T, Heal W, Ter Haar R, Henzler C, Orr HT. O'Callaghan B, et al. Among authors: rainwater o. Mol Ther Nucleic Acids. 2020 Sep 4;21:1006-1016. doi: 10.1016/j.omtn.2020.07.030. Epub 2020 Jul 25. Mol Ther Nucleic Acids. 2020. PMID: 32818920 Free PMC article.
15 results