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203 results

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Page 1
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: roma g. Nat Commun. 2022 Mar 3;13(1):1150. doi: 10.1038/s41467-022-28653-6. Nat Commun. 2022. PMID: 35241644 Free PMC article.
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: roma g. Nat Chem Biol. 2015 Jul;11(7):511-7. doi: 10.1038/nchembio.1837. Epub 2015 Jun 1. Nat Chem Biol. 2015. PMID: 26030728
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: roma g. 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: roma g. Nat Chem Biol. 2016 Apr;12(4):304. doi: 10.1038/nchembio0416-304c. Nat Chem Biol. 2016. PMID: 26991088 No abstract available.
MiR-210 promotes sensory hair cell formation in the organ of corti.
Riccardi S, Bergling S, Sigoillot F, Beibel M, Werner A, Leighton-Davies J, Knehr J, Bouwmeester T, Parker CN, Roma G, Kinzel B. Riccardi S, et al. Among authors: roma g. BMC Genomics. 2016 Apr 27;17:309. doi: 10.1186/s12864-016-2620-7. BMC Genomics. 2016. PMID: 27121005 Free PMC article.
Functional CRISPR screening identifies the ufmylation pathway as a regulator of SQSTM1/p62.
DeJesus R, Moretti F, McAllister G, Wang Z, Bergman P, Liu S, Frias E, Alford J, Reece-Hoyes JS, Lindeman A, Kelliher J, Russ C, Knehr J, Carbone W, Beibel M, Roma G, Ng A, Tallarico JA, Porter JA, Xavier RJ, Mickanin C, Murphy LO, Hoffman GR, Nyfeler B. DeJesus R, et al. Among authors: roma g. Elife. 2016 Jun 28;5:e17290. doi: 10.7554/eLife.17290. Elife. 2016. PMID: 27351204 Free PMC article.
CGG Repeat-Induced FMR1 Silencing Depends on the Expansion Size in Human iPSCs and Neurons Carrying Unmethylated Full Mutations.
Brykczynska U, Pecho-Vrieseling E, Thiemeyer A, Klein J, Fruh I, Doll T, Manneville C, Fuchs S, Iazeolla M, Beibel M, Roma G, Naumann U, Kelley N, Oakeley EJ, Mueller M, Gomez-Mancilla B, Bühler M, Tabolacci E, Chiurazzi P, Neri G, Bouwmeester T, Di Giorgio FP, Fodor BD. Brykczynska U, et al. Among authors: roma g. Stem Cell Reports. 2016 Dec 13;7(6):1059-1071. doi: 10.1016/j.stemcr.2016.10.004. Epub 2016 Nov 10. Stem Cell Reports. 2016. PMID: 27840045 Free PMC article.
Screening of Intestinal Crypt Organoids: A Simple Readout for Complex Biology.
Ley S, Galuba O, Salathe A, Melin N, Aebi A, Pikiolek M, Knehr J, Carbone W, Beibel M, Nigsch F, Roma G, d'Ario G, Kirkland S, Bouchez LC, Gubser Keller C, Bouwmeester T, Parker CN, Ruffner H. Ley S, et al. Among authors: roma g. SLAS Discov. 2017 Jun;22(5):571-582. doi: 10.1177/2472555216683651. Epub 2017 Jan 6. SLAS Discov. 2017. PMID: 28345372 Free article.
203 results