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Page 1
Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.
Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M. Liu G, et al. Among authors: moon yc. Hum Mol Genet. 2024 Jan 20;33(3):211-223. doi: 10.1093/hmg/ddad173. Hum Mol Genet. 2024. PMID: 37819629 Free PMC article.
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.
Cao L, Weetall M, Trotta C, Cintron K, Ma J, Kim MJ, Furia B, Romfo C, Graci JD, Li W, Du J, Sheedy J, Hedrick J, Risher N, Yeh S, Qi H, Arasu T, Hwang S, Lennox W, Kong R, Petruska J, Moon YC, Babiak J, Davis TW, Jacobson A, Almstead NG, Branstrom A, Colacino JM, Peltz SW. Cao L, et al. Among authors: moon yc. Mol Cancer Ther. 2019 Jan;18(1):3-16. doi: 10.1158/1535-7163.MCT-18-0863. Epub 2018 Oct 23. Mol Cancer Ther. 2019. PMID: 30352802 Free PMC article.
Small molecule splicing modifiers with systemic HTT-lowering activity.
Bhattacharyya A, Trotta CR, Narasimhan J, Wiedinger KJ, Li W, Effenberger KA, Woll MG, Jani MB, Risher N, Yeh S, Cheng Y, Sydorenko N, Moon YC, Karp GM, Weetall M, Dakka A, Gabbeta V, Naryshkin NA, Graci JD, Tripodi T Jr, Southwell A, Hayden M, Colacino JM, Peltz SW. Bhattacharyya A, et al. Among authors: moon yc. Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z. Nat Commun. 2021. PMID: 34911927 Free PMC article.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Among authors: moon yc. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent.
Jernigan F, Branstrom A, Baird JD, Cao L, Dali M, Furia B, Kim MJ, O'Keefe K, Kong R, Laskin OL, Colacino JM, Pykett M, Mollin A, Sheedy J, Dumble M, Moon YC, Sheridan R, Mühlethaler T, Spiegel RJ, Prota AE, Steinmetz MO, Weetall M. Jernigan F, et al. Among authors: moon yc. Mol Cancer Ther. 2021 Oct;20(10):1846-1857. doi: 10.1158/1535-7163.MCT-20-0774. Epub 2021 Jul 26. Mol Cancer Ther. 2021. PMID: 34315764 Free PMC article. Clinical Trial.
Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy.
Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. Woll MG, et al. Among authors: moon yc. J Med Chem. 2016 Jul 14;59(13):6070-85. doi: 10.1021/acs.jmedchem.6b00460. Epub 2016 Jul 6. J Med Chem. 2016. PMID: 27299569
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
Friesen WJ, Johnson B, Sierra J, Zhuo J, Vazirani P, Xue X, Tomizawa Y, Baiazitov R, Morrill C, Ren H, Babu S, Moon YC, Branstrom A, Mollin A, Hedrick J, Sheedy J, Elfring G, Weetall M, Colacino JM, Welch EM, Peltz SW. Friesen WJ, et al. Among authors: moon yc. PLoS One. 2018 Oct 25;13(10):e0206158. doi: 10.1371/journal.pone.0206158. eCollection 2018. PLoS One. 2018. PMID: 30359426 Free PMC article.
30 results