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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: yang p. 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: yang p. 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.
Mapping SCA1 regional vulnerabilities reveals neural and skeletal muscle contributions to disease.
Duvick L, Southern WM, Benzow KA, Burch ZN, Handler HP, Mitchell JS, Kuivinen H, Gadiparthi U, Yang P, Soles A, Sheeler CA, Rainwater O, Serres S, Lind EB, Nichols-Meade T, You Y, O'Callaghan B, Zoghbi HY, Cvetanovic M, Wheeler VC, Ervasti JM, Koob MD, Orr HT. Duvick L, et al. Among authors: yang p. JCI Insight. 2024 Mar 21;9(9):e176057. doi: 10.1172/jci.insight.176057. JCI Insight. 2024. PMID: 38512434 Free PMC article.
Delineating regional vulnerability in the neurodegenerative disease SCA1 using a conditional mutant ATXN1 mouse.
Duvick L, Southern WM, Benzow K, Burch ZN, Handler HP, Mitchell JS, Kuivinen H, Gadiparthi UK, Yang P, Soles A, Scheeler C, Rainwater O, Serres S, Lind E, Nichols-Meade T, O'Callaghan B, Zoghbi HY, Cvetanovic M, Wheeler VC, Ervasti JM, Koob MD, Orr HT. Duvick L, et al. Among authors: yang p. bioRxiv [Preprint]. 2023 Jun 30:2023.02.08.527710. doi: 10.1101/2023.02.08.527710. bioRxiv. 2023. PMID: 36798410 Free PMC article. Preprint.
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