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Page 1
Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA).
Cheung AK, Hurley B, Kerrigan R, Shu L, Chin DN, Shen Y, O'Brien G, Sung MJ, Hou Y, Axford J, Cody E, Sun R, Fazal A, Fridrich C, Sanchez CC, Tomlinson RC, Jain M, Deng L, Hoffmaster K, Song C, Van Hoosear M, Shin Y, Servais R, Towler C, Hild M, Curtis D, Dietrich WF, Hamann LG, Briner K, Chen KS, Kobayashi D, Sivasankaran R, Dales NA. Cheung AK, et al. Among authors: hild m. J Med Chem. 2018 Dec 27;61(24):11021-11036. doi: 10.1021/acs.jmedchem.8b01291. Epub 2018 Dec 13. J Med Chem. 2018. PMID: 30407821
SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Palacino J, Swalley SE, Song C, Cheung AK, Shu L, Zhang X, Van Hoosear M, Shin Y, Chin DN, Keller CG, Beibel M, Renaud NA, Smith TM, Salcius M, Shi X, Hild M, Servais R, Jain M, Deng L, Bullock C, McLellan M, Schuierer S, Murphy L, Blommers MJ, Blaustein C, Berenshteyn F, Lacoste A, Thomas JR, Roma G, Michaud GA, Tseng BS, Porter JA, Myer VE, Tallarico JA, Hamann LG, Curtis D, Fishman MC, Dietrich WF, Dales NA, Sivasankaran R. Palacino J, et al. Among authors: hild m. Nat Chem Biol. 2015 Jul;11(7):511-7. doi: 10.1038/nchembio.1837. Epub 2015 Jun 1. Nat Chem Biol. 2015. PMID: 26030728
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Keller CG, Shin Y, Monteys AM, Renaud N, Beibel M, Teider N, Peters T, Faller T, St-Cyr S, Knehr J, Roma G, Reyes A, Hild M, Lukashev D, Theil D, Dales N, Cha JH, Borowsky B, Dolmetsch R, Davidson BL, Sivasankaran R. Keller CG, et al. Among authors: hild m. Nat Commun. 2022 Mar 3;13(1):1150. doi: 10.1038/s41467-022-28653-6. Nat Commun. 2022. PMID: 35241644 Free PMC article.
Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Palacino J, Swalley SE, Song C, Cheung AK, Shu L, Zhang X, Van Hoosear M, Shin Y, Chin DN, Keller CG, Beibel M, Renaud NA, Smith TM, Salcius M, Shi X, Hild M, Servais R, Jain M, Deng L, Bullock C, McLellan M, Schuierer S, Murphy L, Blommers MJ, Blaustein C, Berenshteyn F, Lacoste A, Thomas JR, Roma G, Michaud GA, Tseng BS, Porter JA, Myer VE, Tallarico JA, Hamann LG, Curtis D, Fishman MC, Dietrich WF, Dales NA, Sivasankaran R. Palacino J, et al. Among authors: hild m. Nat Chem Biol. 2015 Sep;11(9):741. doi: 10.1038/nchembio0915-741a. Nat Chem Biol. 2015. PMID: 26284678 No abstract available.
Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Palacino J, Swalley SE, Song C, Cheung AK, Shu L, Zhang X, Van Hoosear M, Shin Y, Chin DN, Keller CG, Beibel M, Renaud NA, Smith TM, Salcius M, Shi X, Hild M, Servais R, Jain M, Deng L, Bullock C, McLellan M, Schuierer S, Murphy L, Blommers MJ, Blaustein C, Berenshteyn F, Lacoste A, Thomas JR, Roma G, Michaud GA, Tseng BS, Porter JA, Myer VE, Tallarico JA, Hamann LG, Curtis D, Fishman MC, Dietrich WF, Dales NA, Sivasankaran R. Palacino J, et al. Among authors: hild m. Nat Chem Biol. 2016 Apr;12(4):304. doi: 10.1038/nchembio0416-304c. Nat Chem Biol. 2016. PMID: 26991088 No abstract available.
Tankyrase inhibition stabilizes axin and antagonizes Wnt signalling.
Huang SM, Mishina YM, Liu S, Cheung A, Stegmeier F, Michaud GA, Charlat O, Wiellette E, Zhang Y, Wiessner S, Hild M, Shi X, Wilson CJ, Mickanin C, Myer V, Fazal A, Tomlinson R, Serluca F, Shao W, Cheng H, Shultz M, Rau C, Schirle M, Schlegl J, Ghidelli S, Fawell S, Lu C, Curtis D, Kirschner MW, Lengauer C, Finan PM, Tallarico JA, Bouwmeester T, Porter JA, Bauer A, Cong F. Huang SM, et al. Among authors: hild m. Nature. 2009 Oct 1;461(7264):614-20. doi: 10.1038/nature08356. Epub 2009 Sep 16. Nature. 2009. PMID: 19759537
DRUG-seq for miniaturized high-throughput transcriptome profiling in drug discovery.
Ye C, Ho DJ, Neri M, Yang C, Kulkarni T, Randhawa R, Henault M, Mostacci N, Farmer P, Renner S, Ihry R, Mansur L, Keller CG, McAllister G, Hild M, Jenkins J, Kaykas A. Ye C, et al. Among authors: hild m. Nat Commun. 2018 Oct 17;9(1):4307. doi: 10.1038/s41467-018-06500-x. Nat Commun. 2018. PMID: 30333485 Free PMC article.
Structure of the DDB1-CRBN E3 ubiquitin ligase in complex with thalidomide.
Fischer ES, Böhm K, Lydeard JR, Yang H, Stadler MB, Cavadini S, Nagel J, Serluca F, Acker V, Lingaraju GM, Tichkule RB, Schebesta M, Forrester WC, Schirle M, Hassiepen U, Ottl J, Hild M, Beckwith RE, Harper JW, Jenkins JL, Thomä NH. Fischer ES, et al. Among authors: hild m. Nature. 2014 Aug 7;512(7512):49-53. doi: 10.1038/nature13527. Epub 2014 Jul 16. Nature. 2014. PMID: 25043012 Free PMC article.
DRUG-seq Provides Unbiased Biological Activity Readouts for Neuroscience Drug Discovery.
Li J, Ho DJ, Henault M, Yang C, Neri M, Ge R, Renner S, Mansur L, Lindeman A, Kelly B, Tumkaya T, Ke X, Soler-Llavina G, Shanker G, Russ C, Hild M, Gubser Keller C, Jenkins JL, Worringer KA, Sigoillot FD, Ihry RJ. Li J, et al. Among authors: hild m. ACS Chem Biol. 2022 Jun 17;17(6):1401-1414. doi: 10.1021/acschembio.1c00920. Epub 2022 May 4. ACS Chem Biol. 2022. PMID: 35508359 Free PMC article.
64 results