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Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.
Roy B, Friesen WJ, Tomizawa Y, Leszyk JD, Zhuo J, Johnson B, Dakka J, Trotta CR, Xue X, Mutyam V, Keeling KM, Mobley JA, Rowe SM, Bedwell DM, Welch EM, Jacobson A. Roy B, et al. Among authors: rowe sm. Proc Natl Acad Sci U S A. 2016 Nov 1;113(44):12508-12513. doi: 10.1073/pnas.1605336113. Epub 2016 Oct 4. Proc Natl Acad Sci U S A. 2016. PMID: 27702906 Free PMC article.
Synthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftor.
Xue X, Mutyam V, Tang L, Biswas S, Du M, Jackson LA, Dai Y, Belakhov V, Shalev M, Chen F, Schacht J, J Bridges R, Baasov T, Hong J, Bedwell DM, Rowe SM. Xue X, et al. Among authors: rowe sm. Am J Respir Cell Mol Biol. 2014 Apr;50(4):805-16. doi: 10.1165/rcmb.2013-0282OC. Am J Respir Cell Mol Biol. 2014. PMID: 24251786 Free PMC article.
Characterization of defects in ion transport and tissue development in cystic fibrosis transmembrane conductance regulator (CFTR)-knockout rats.
Tuggle KL, Birket SE, Cui X, Hong J, Warren J, Reid L, Chambers A, Ji D, Gamber K, Chu KK, Tearney G, Tang LP, Fortenberry JA, Du M, Cadillac JM, Bedwell DM, Rowe SM, Sorscher EJ, Fanucchi MV. Tuggle KL, et al. Among authors: rowe sm. PLoS One. 2014 Mar 7;9(3):e91253. doi: 10.1371/journal.pone.0091253. eCollection 2014. PLoS One. 2014. PMID: 24608905 Free PMC article.
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group. Kerem E, et al. Among authors: rowe sm. Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15. Lancet Respir Med. 2014. PMID: 24836205 Free PMC article. Clinical Trial.
Discovery of Clinically Approved Agents That Promote Suppression of Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations.
Mutyam V, Du M, Xue X, Keeling KM, White EL, Bostwick JR, Rasmussen L, Liu B, Mazur M, Hong JS, Falk Libby E, Liang F, Shang H, Mense M, Suto MJ, Bedwell DM, Rowe SM. Mutyam V, et al. Among authors: rowe sm. Am J Respir Crit Care Med. 2016 Nov 1;194(9):1092-1103. doi: 10.1164/rccm.201601-0154OC. Am J Respir Crit Care Med. 2016. PMID: 27104944 Free PMC article.
328 results