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Page 1
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
Self-renewal as a therapeutic target in human colorectal cancer.
Kreso A, van Galen P, Pedley NM, Lima-Fernandes E, Frelin C, Davis T, Cao L, Baiazitov R, Du W, Sydorenko N, Moon YC, Gibson L, Wang Y, Leung C, Iscove NN, Arrowsmith CH, Szentgyorgyi E, Gallinger S, Dick JE, O'Brien CA. Kreso A, et al. Among authors: moon yc. Nat Med. 2014 Jan;20(1):29-36. doi: 10.1038/nm.3418. Epub 2013 Dec 1. Nat Med. 2014. PMID: 24292392
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.
Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F. Naryshkin NA, et al. Among authors: moon yc. Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127. Science. 2014. PMID: 25104390
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
BMI-1 Targeting Interferes with Patient-Derived Tumor-Initiating Cell Survival and Tumor Growth in Prostate Cancer.
Bansal N, Bartucci M, Yusuff S, Davis S, Flaherty K, Huselid E, Patrizii M, Jones D, Cao L, Sydorenko N, Moon YC, Zhong H, Medina DJ, Kerrigan J, Stein MN, Kim IY, Davis TW, DiPaola RS, Bertino JR, Sabaawy HE. Bansal N, et al. Among authors: moon yc. Clin Cancer Res. 2016 Dec 15;22(24):6176-6191. doi: 10.1158/1078-0432.CCR-15-3107. Epub 2016 Jun 15. Clin Cancer Res. 2016. PMID: 27307599 Free PMC article.
Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.
Weetall M, Davis T, Elfring G, Northcutt V, Cao L, Moon YC, Riebling P, Dali M, Hirawat S, Babiak J, Colacino J, Almstead N, Spiegel R, Peltz SW. Weetall M, et al. Among authors: moon yc. Clin Pharmacol Drug Dev. 2016 Jul;5(4):296-305. doi: 10.1002/cpdd.240. Epub 2016 Feb 2. Clin Pharmacol Drug Dev. 2016. PMID: 27310330 Free PMC article. Clinical Trial.
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform.
Cao L, Weetall M, Bombard J, Qi H, Arasu T, Lennox W, Hedrick J, Sheedy J, Risher N, Brooks PC, Trifillis P, Trotta C, Moon YC, Babiak J, Almstead NG, Colacino JM, Davis TW, Peltz SW. Cao L, et al. Among authors: moon yc. PLoS One. 2016 Dec 16;11(12):e0168366. doi: 10.1371/journal.pone.0168366. eCollection 2016. PLoS One. 2016. PMID: 27992500 Free PMC article.
The nucleoside analog clitocine is a potent and efficacious readthrough agent.
Friesen WJ, Trotta CR, Tomizawa Y, Zhuo J, Johnson B, Sierra J, Roy B, Weetall M, Hedrick J, Sheedy J, Takasugi J, Moon YC, Babu S, Baiazitov R, Leszyk JD, Davis TW, Colacino JM, Peltz SW, Welch EM. Friesen WJ, et al. Among authors: moon yc. RNA. 2017 Apr;23(4):567-577. doi: 10.1261/rna.060236.116. Epub 2017 Jan 17. RNA. 2017. PMID: 28096517 Free PMC article.
30 results