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Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA).
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: van hoosear 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
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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: van hoosear m.
Nat Chem Biol. 2015 Sep;11(9):741. doi: 10.1038/nchembio0915-741a.
Nat Chem Biol. 2015.
PMID: 26284678
No abstract available.
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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: van hoosear m.
Nat Chem Biol. 2016 Apr;12(4):304. doi: 10.1038/nchembio0416-304c.
Nat Chem Biol. 2016.
PMID: 26991088
No abstract available.
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Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ.
Song C, et al. Among authors: van hoosear m.
Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168.
Hum Gene Ther Clin Dev. 2018.
PMID: 30280954
Free PMC article.
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