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Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.
Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J, Hazlewood A, Zhou J, McCartney J, Arumugam V, Decker C, Yang J, Young C, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu P. Van Goor F, et al. Among authors: negulescu p. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18825-30. doi: 10.1073/pnas.0904709106. Epub 2009 Oct 21. Proc Natl Acad Sci U S A. 2009. PMID: 19846789 Free PMC article.
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.
Van Goor F, Hadida S, Grootenhuis PD, Burton B, Stack JH, Straley KS, Decker CJ, Miller M, McCartney J, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu PA. Van Goor F, et al. Among authors: negulescu pa. Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18843-8. doi: 10.1073/pnas.1105787108. Epub 2011 Oct 5. Proc Natl Acad Sci U S A. 2011. PMID: 21976485 Free PMC article.
Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules.
Van Goor F, Straley KS, Cao D, González J, Hadida S, Hazlewood A, Joubran J, Knapp T, Makings LR, Miller M, Neuberger T, Olson E, Panchenko V, Rader J, Singh A, Stack JH, Tung R, Grootenhuis PD, Negulescu P. Van Goor F, et al. Among authors: negulescu p. Am J Physiol Lung Cell Mol Physiol. 2006 Jun;290(6):L1117-30. doi: 10.1152/ajplung.00169.2005. Epub 2006 Jan 27. Am J Physiol Lung Cell Mol Physiol. 2006. PMID: 16443646 Free article.
G970R-CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect.
Fidler MC, Buckley A, Sullivan JC, Statia M, Boj SF, Vries RGJ, Munck A, Higgins M, Moretto Zita M, Negulescu P, van Goor F, De Boeck K. Fidler MC, et al. Among authors: negulescu p. Clin Transl Sci. 2021 Mar;14(2):656-663. doi: 10.1111/cts.12927. Epub 2020 Dec 6. Clin Transl Sci. 2021. PMID: 33278322 Free PMC article. Clinical Trial.
In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis.
Sun X, Yi Y, Yan Z, Rosen BH, Liang B, Winter MC, Evans TIA, Rotti PG, Yang Y, Gray JS, Park SY, Zhou W, Zhang Y, Moll SR, Woody L, Tran DM, Jiang L, Vonk AM, Beekman JM, Negulescu P, Van Goor F, Fiorino DF, Gibson-Corley KN, Engelhardt JF. Sun X, et al. Among authors: negulescu p. Sci Transl Med. 2019 Mar 27;11(485):eaau7531. doi: 10.1126/scitranslmed.aau7531. Sci Transl Med. 2019. PMID: 30918114 Free PMC article.
43 results