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α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.
Cole TA, Zhao H, Collier TJ, Sandoval I, Sortwell CE, Steece-Collier K, Daley BF, Booms A, Lipton J, Welch M, Berman M, Jandreski L, Graham D, Weihofen A, Celano S, Schulz E, Cole-Strauss A, Luna E, Quach D, Mohan A, Bennett CF, Swayze EE, Kordasiewicz HB, Luk KC, Paumier KL. Cole TA, et al. JCI Insight. 2021 Mar 8;6(5):e135633. doi: 10.1172/jci.insight.135633. JCI Insight. 2021. PMID: 33682798 Free PMC article.
MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.
Hoye ML, Koval ED, Wegener AJ, Hyman TS, Yang C, O'Brien DR, Miller RL, Cole T, Schoch KM, Shen T, Kunikata T, Richard JP, Gutmann DH, Maragakis NJ, Kordasiewicz HB, Dougherty JD, Miller TM. Hoye ML, et al. J Neurosci. 2017 May 31;37(22):5574-5586. doi: 10.1523/JNEUROSCI.3582-16.2017. Epub 2017 Apr 17. J Neurosci. 2017. PMID: 28416596 Free PMC article.
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.
McCampbell A, Cole T, Wegener AJ, Tomassy GS, Setnicka A, Farley BJ, Schoch KM, Hoye ML, Shabsovich M, Sun L, Luo Y, Zhang M, Comfort N, Wang B, Amacker J, Thankamony S, Salzman DW, Cudkowicz M, Graham DL, Bennett CF, Kordasiewicz HB, Swayze EE, Miller TM. McCampbell A, et al. J Clin Invest. 2018 Aug 1;128(8):3558-3567. doi: 10.1172/JCI99081. Epub 2018 Jul 16. J Clin Invest. 2018. PMID: 30010620 Free PMC article.
Protein production is an early biomarker for RNA-targeted therapies.
Self WK, Schoch KM, Alex J, Barthélemy N, Bollinger JG, Sato C, Cole T, Kordasiewicz HB, Swayze E, Bateman RJ, Miller TM. Self WK, et al. Ann Clin Transl Neurol. 2018 Oct 14;5(12):1492-1504. doi: 10.1002/acn3.657. eCollection 2018 Dec. Ann Clin Transl Neurol. 2018. PMID: 30564616 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. 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.
Red blood cells are the major source of alpha-synuclein in blood.
Barbour R, Kling K, Anderson JP, Banducci K, Cole T, Diep L, Fox M, Goldstein JM, Soriano F, Seubert P, Chilcote TJ. Barbour R, et al. Neurodegener Dis. 2008;5(2):55-9. doi: 10.1159/000112832. Epub 2008 Jan 4. Neurodegener Dis. 2008. PMID: 18182779
30 results