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Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
Open accessAbstract
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator ( CFTR ) gene that impair the function of CFTR, an epithelial chloride channel required for proper function of the lung, pancreas, and other organs. Most patients with CF carry the F508del CFTR mutation, which causes defective CFTR protein folding and processing in the endoplasmic reticulum, resulting in minimal amounts of CFTR at the cell surface. One strategy to treat these patients is to correct the processing of F508del-CFTR with small molecules. Here we describe the in vitro pharmacology of VX-809, a CFTR corrector that was advanced into clinical development for the treatment of CF. In cultured human bronchial epithelial cells isolated from patients with CF homozygous for F508del, VX-809 improved F508del-CFTR processing in the endoplasmic reticulum and enhanced chloride secretion to approximately 14% of non-CF human bronchial epithelial cells (EC 50 , 81 ± 19 nM), a level associated with mild CF in patients with less disruptive CFTR mutations. F508del-CFTR corrected by VX-809 exhibited biochemical and functional characteristics similar to normal CFTR, including biochemical susceptibility to proteolysis, residence time in the plasma membrane, and single-channel open probability. VX-809 was more efficacious and selective for CFTR than previously reported CFTR correctors. VX-809 represents a class of CFTR corrector that specifically addresses the underlying processing defect in F508del-CFTR.
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“Mutations in CFTR cause multi-organ dysfunctions affecting lungs, pancreas, liver and intestine187.”
From Organoids (2022) · cites this paper as [187]
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Cites
Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA
Riordan, J. R., Rommens, J. M., Kerem, B.-S., Alon, N., Rozmahel, R., Grzelczak, Z., Zielenski, J., Lok, S., Plavsic, N., Chou, J.-L., et al. (1989) · Science · doi:10.1126/science.2475911 · Cited by 7,418
Cheng, S. H., Gregory, R. J., Marshall, J., Paul, S., Souza, D. W., White, G. A., O’Riordan, C. R., & Smith, A. E. (1990) · Cell · doi:10.1016/0092-8674(90)90148-8 · Cited by 1,792
Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive
Denning, G. M., Anderson, M. P., Amara, J. F., Marshall, J., Smith, A. E., & Welsh, M. J. (1992) · Nature · doi:10.1038/358761a0 · Cited by 1,236
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
Goor, F. V., Hadidaꝉ, S., Grootenhuis, P. D. J., Burton, B., Cao, D., Neuberger, T., Turnbull, A., Singh, A. K., Joubran, J., Hazlewood, A., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0904709106 · Cited by 1,225
Self-citationFarrell, P. M., Rosenstein, B. J., White, T. B., Accurso, F. J., Castellani, C., Cutting, G. R., Durie, P. R., LeGrys, V. A., Massie, J., Parad, R. B., et al. (2008) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2008.05.005 · Cited by 1,093
Bobadilla, J. L., Macek, M., Fine, J. P., & Farrell, P. M. (2002) · Human Mutation · doi:10.1002/humu.10041 · Cited by 1,032
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation
Accurso, F. J., Rowe, S. M., Clancy, J. P., Boyle, M., Dunitz, J., Durie, P. R., Sagel, S. D., Hornick, D. B., Konstan, M. W., Donaldson, S. H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa0909825 · Cited by 803
Self-citationAltered chloride ion channel kinetics associated with the ΔF508 cystic fibrosis mutation
Dalemans, W., Barbry, P., Champigny, G., Jallat, S., Jallat, S., Dott, K., Dreyer, D., Crystal, R. G., Pavirani, A., Lecocq, J.-P., et al. (1991) · Nature · doi:10.1038/354526a0 · Cited by 707
Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice
Castellani, C., Cuppens, H., Maçek, M., Cassiman, J. J., Kerem, E., Durie, P. R., Tullis, E. D., Assael, B. M., Bombieri, C., Brown, A., et al. (2008) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2008.03.009 · Cited by 624
Pedemonte, N. (2005) · Journal of Clinical Investigation · doi:10.1172/jci24898 · Cited by 551
Research works printed but not linked: 1
- ; Cystic Fibrosis Foundation US Cystic Fibrosis Patient Registry (Cystic Fibrosis Foundation, Bethesda, 2002).
Cited by
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Items 501–600 of 1,000
Fukuda, R., & Okiyoneda, T. (2020) · Pharmaceuticals · doi:10.3390/ph13040075 · Cited by 23
Cystic Fibrosis Lung Disease in the Aging Population
Künzi, L., Easter, M., Hirsch, M. J., & Krick, S. (2021) · Frontiers in Pharmacology · doi:10.3389/fphar.2021.601438 · Cited by 26
Luettich, K., Sharma, M., Yepiskoposyan, H., Breheny, D., & Lowe, F. (2021) · Frontiers in Toxicology · doi:10.3389/ftox.2021.750254 · Cited by 23
Honrubia, J. M., Gutierrez-Alvarez, F. J., Sanz‐Bravo, A., González-Miranda, E., Muñoz‐Santos, D., Castaño-Rodríguez, C., Wang, L., Villarejo-Torres, M., Ripoll-Gómez, J., Esteban, A., et al. (2023) · mBio · doi:10.1128/mbio.03136-22 · Cited by 23
Cystic fibrosis transmembrane conductance regulator dysfunction and its treatment
Hull, J. (2012) · Journal of the Royal Society of Medicine · doi:10.1258/jrsm.2012.12s001 · Cited by 25
Advances in the Diagnosis and Treatment of Cystic Fibrosis
Martiniano, S. L., Hoppe, J. E., Sagel, S. D., & Zemanick, E. T. (2014) · Advances in Pediatrics · doi:10.1016/j.yapd.2014.03.002 · Cited by 25
Loo, T. W., Bartlett, M. C., Shi, L., & Clarke, D. M. (2011) · Biochemical Pharmacology · doi:10.1016/j.bcp.2011.11.014 · Cited by 23
Namkung, W., Park, J., Seo, Y., & Verkman, A. S. (2013) · Molecular Pharmacology · doi:10.1124/mol.113.086348 · Cited by 23
Stem cell‐derived organoids to model gastrointestinal facets of cystic fibrosis
Hohwieler, M., Perkhofer, L., Liebau, S., Seufferlein, T. T. W., Müller, M., Illing, A., & Kleger, A. (2016) · United European Gastroenterology Journal · doi:10.1177/2050640616670565 · Cited by 22
Ligand-promoted protein folding by biased kinetic partitioning
Hingorani, K. S., Metcalf, M. C., Deming, D., Garman, S. C., Powers, E. T., & Gierasch, L. M. (2017) · Nature Chemical Biology · doi:10.1038/nchembio.2303 · Cited by 23
Kotha, K. S., Szczesniak, R. D., Naren, A. P., Fenchel, M. C., Duan, L. L., McPhail, G. L., & Clancy, J. P. (2015) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2015.07.002 · Cited by 25
Translational research to enable personalized treatment of cystic fibrosis
Hagemeijer, M. C., Siegwart, D. J., Strug, L. J., Cebotaru, L., Torres, M. J., Sofoluwe, A., & Beekman, J. M. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.10.017 · Cited by 22
Evans, I. A., Sun, X., Liang, B., Vegter, A. R., Guo, L., Lynch, T. J., Zhang, Y., Zhang, Y., Yi, Y., Yu, Y., et al. (2024) · JCI Insight · doi:10.1172/jci.insight.157229 · Cited by 22
Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor
Tümmler, B. (2023) · Frontiers in Pharmacology · doi:10.3389/fphar.2023.1158207 · Cited by 22
Vachel, L., Norez, C., Jayle, C., Becq, F., & Vandebrouck, C. (2014) · Cell Calcium · doi:10.1016/j.ceca.2014.11.005 · Cited by 22
Brewington, J. J., McPhail, G. L., & Clancy, J. P. (2015) · Expert Review of Respiratory Medicine · doi:10.1586/17476348.2016.1122527 · Cited by 24
Transformative therapies for rare CFTR missense alleles
Oliver, K. E., Han, S. T., Sorscher, E. J., & Cutting, G. R. (2017) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2017.09.018 · Cited by 23
Ponzano, S., Nigrelli, G., Fregonese, L., Eichler, I., Bertozzi, F., Bandiera, T., Galietta, L. J. V., & Papaluca, M. (2018) · European Respiratory Review · doi:10.1183/16000617.0124-2017 · Cited by 23
Yanda, M. K., Cha, B., Cebotaru, C. V., & Cebotaru, L. (2019) · Journal of Biological Chemistry · doi:10.1074/jbc.ra119.010320 · Cited by 22
Tang, Y., Yan, Z., Lin, S., Huntemann, E. D., Feng, Z., Park, S.-Y., Sun, X., Yuen, E., & Engelhardt, J. F. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.008 · Cited by 21
Guanylate cyclase 2C agonism corrects CFTR mutants
Arora, K., Huang, Y., Mun, K., Yarlagadda, S., Sundaram, N., Kessler, M. M., Hannig, G., Kurtz, C. B., Silos‐Santiago, I., Helmrath, M. A., et al. (2017) · JCI Insight · doi:10.1172/jci.insight.93686 · Cited by 20
Tassini, S., Langron, E., Delang, L., Mirabelli, C., Lanko, K., Crespan, E., Kiššová, M., Tagliavini, G., Fontò, G., Bertoni, S., et al. (2019) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.9b01416 · Cited by 21
CFTR: New insights into structure and function and implications for modulation by small molecules
Kleizen, B., Hunt, J. F., Callebaut, I., Hwang, T., Sermet‐Gaudelus, I., Hafkemeyer, S., & Sheppard, D. N. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.10.021 · Cited by 22
Role of Protein Kinase A-Mediated Phosphorylation in CFTR Channel Activity Regulation
Sala, A. D., Prono, G., Hirsch, E., & Ghigo, A. (2021) · Frontiers in Physiology · doi:10.3389/fphys.2021.690247 · Cited by 21
Bui, S., Masson, A., Enaud, R., Roditis, L., Dournes, G., Galodé, F., Collet, C., Mas, E., Languepin, J., Fayon, M. J., et al. (2021) · Frontiers in Pediatrics · doi:10.3389/fped.2021.744705 · Cited by 21
Cystic fibrosis — What are the prospects for a cure?
Kumar, S., Tana, A., & Shankar, A. (2014) · European Journal of Internal Medicine · doi:10.1016/j.ejim.2014.09.018 · Cited by 24
Bithiazole Correctors Rescue CFTR Mutants by Two Different Mechanisms
Loo, T. W., Bartlett, M. C., & Clarke, D. M. (2013) · Biochemistry · doi:10.1021/bi4008758 · Cited by 21
Blocking protein quality control to counter hereditary cancers
Kampmeyer, C., Nielsen, S. V., Clausen, L., Stein, A., Gerdes, A., Lindorff‐Larsen, K., & Hartmann‐Petersen, R. (2017) · Genes Chromosomes and Cancer · doi:10.1002/gcc.22487 · Cited by 21
Unraveling the Function of Lemur Tyrosine Kinase 2 Network
Cruz, D. F., Farinha, C. M., & Swiatecka‐Urban, A. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00024 · Cited by 21
Tong, K., Barker, D., France, M. W., Burr, L. D., Greville, H. W., Visser, S. K., Middleton, P. G., Wainwright, C. E., Dorahy, D. J., & Wark, P. A. B. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.12.006 · Cited by 21
Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment
Lee, T. N. G., Cholon, D. M., Quinney, N. L., Gentzsch, M., & Esther, C. R. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.04.010 · Cited by 21
Inhibition of Pseudomonas aeruginosa Alginate Synthesis by Ebselen Oxide and Its Analogues
Kim, S.-K., Ngo, H., Dennis, E. K., Chandrika, N. T., DeShong, P. R., Garneau‐Tsodikova, S., & Lee, V. T. (2021) · ACS Infectious Diseases · doi:10.1021/acsinfecdis.1c00045 · Cited by 23
Automated acquisition and analysis of airway surface liquid height by confocal microscopy
Choi, H.-C., Kim, C. S. K., & Tarran, R. (2015) · American Journal of Physiology-Lung Cellular and Molecular Physiology · doi:10.1152/ajplung.00027.2015 · Cited by 21
Gong, X., Liao, Y., Ahner, A., Larsen, M. B., Wang, X., Bertrand, C. A., & Frizzell, R. A. (2018) · Molecular Biology of the Cell · doi:10.1091/mbc.e18-04-0252 · Cited by 21
Self-citationThe future of CFTR modulating therapies for cystic fibrosis
Davies, J. C. (2015) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000000211 · Cited by 20
Chronic β2AR stimulation limits CFTR activation in human airway epithelia
Brewington, J. J., Backstrom, J., Feldman, A., Kramer, E. L., Moncivaiz, J. D., Ostmann, A. J., Zhu, X., Lu, L. J., & Clancy, J. P. (2018) · JCI Insight · doi:10.1172/jci.insight.93029 · Cited by 22
Liu, J., Berg, A. P., Wang, Y., Jantarajit, W., Sutcliffe, K. J., Stevens, E. B., Cao, L., Pregel, M. J., & Sheppard, D. N. (2021) · British Journal of Pharmacology · doi:10.1111/bph.15709 · Cited by 20
Structural Comparative Modeling of Multi-Domain F508del CFTR
McDonald, E. F., Woods, H., Smith, S. T., Kim, M., Schoeder, C. T., Plate, L., & Meiler, J. (2022) · Biomolecules · doi:10.3390/biom12030471 · Cited by 19
Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis
Birimberg‐Schwartz, L., Ip, W., Bartlett, C., Avolio, J., Vonk, A. M., Gunawardena, T. N. A., Du, K., Esmaeili, M., Beekman, J. M., Rommens, J. M., et al. (2023) · Life Science Alliance · doi:10.26508/lsa.202201857 · Cited by 20
Allegretta, C., Difonzo, G., Caponio, F., Tamma, G., & Laselva, O. (2023) · Cells · doi:10.3390/cells12131764 · Cited by 20
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Burgener, E. B., & Cornfield, D. N. (2023) · PEDIATRICS · doi:10.1542/peds.2023-062985 · Cited by 21
Liu, Z., Borlak, J. T., & Tong, W. (2014) · Genome Medicine · doi:10.1186/s13073-014-0094-2 · Cited by 20
Rocca, J., Manin, S., Lesne-Hulin, A., Aissat, A., Verbecq‐Morlot, W., Prulière-Escabasse, V., Wohlhuter‐Haddad, A., Epaud, R., Fanen, P., & Tarze, A. (2016) · British Journal of Pharmacology · doi:10.1111/bph.13464 · Cited by 20
Puglia, M., Landi, C., Gagliardi, A., Breslin, L., Armini, A., Brunetti, J., Pini, A., Bianchi, L., & Bini, L. (2017) · Journal of Proteomics · doi:10.1016/j.jprot.2017.09.013 · Cited by 20
Advances in the Diagnosis and Management of Cystic Fibrosis in the Genomic Era
Wiencek, J. R., & Lo, S. F. (2018) · Clinical Chemistry · doi:10.1373/clinchem.2017.274670 · Cited by 23
Berg, A. P., Hallowell, S., Tibbetts, M., Beasley, C., Brown-Phillips, T., Healy, A., Pustilnik, L. R., Doyonnas, R., & Pregel, M. J. (2019) · SLAS DISCOVERY · doi:10.1177/2472555219849375 · Cited by 20
Matos, A. M., Pinto, F. R., Barros, P., Amaral, M. D., Pepperkok, R., & Matos, P. (2019) · Journal of Biological Chemistry · doi:10.1074/jbc.ra119.008738 · Cited by 21
Lipid-driven CFTR clustering is impaired in cystic fibrosis and restored by corrector drugs
Abu‐Arish, A., Pandžić, E., Luo, Y., Sato, Y., Turner, M. J., Wiseman, P. W., & Hanrahan, J. W. (2022) · Journal of Cell Science · doi:10.1242/jcs.259002 · Cited by 20
Cebotaru, L., Woodward, O. M., Cebotaru, V., & Guggino, W. B. (2013) · Journal of Biological Chemistry · doi:10.1074/jbc.m112.420489 · Cited by 20
Pharmacological rescue of mutant CFTR protein improves the viscoelastic properties of CF mucus
Gianotti, A., Capurro, V., Scudieri, P., Galietta, L. J. V., Morán, Ó., & Zegarra‐Moran, O. (2015) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2015.11.003 · Cited by 21
Laselva, O., Erwood, S., Du, K., Ivakine, E. A., & Bear, C. E. (2019) · FASEB BioAdvances · doi:10.1096/fba.2019-00039 · Cited by 19
Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis
Haq, I., Almulhem, M. M., Soars, S., Poulton, D., & Brodlie, M. (2022) · Pharmacogenomics and Personalized Medicine · doi:10.2147/pgpm.s245603 · Cited by 20
Ocular development after highly effective modulator treatment early in life
Zhu, Y., Li, D., Reyes‐Ortega, F., Chinnery, H. R., & Schneider‐Futschik, E. K. (2023) · Frontiers in Pharmacology · doi:10.3389/fphar.2023.1265138 · Cited by 19
Functional Rescue of a Kidney Anion Exchanger 1 Trafficking Mutant in Renal Epithelial Cells
Chu, C., King, J. C., Berrini, M., Alexander, R. T., & Cordat, E. (2013) · PLoS ONE · doi:10.1371/journal.pone.0057062 · Cited by 19
Liu, J., Bihler, H. J., Farinha, C. M., Awatade, N. T., Romão, A. M., Mercadante, D. L., Cheng, Y., Musisi, I., Jantarajit, W., Wang, Y., et al. (2018) · British Journal of Pharmacology · doi:10.1111/bph.14141 · Cited by 19
Investigating Potassium Channels in Budding Yeast: A Genetic Sandbox.
Mackie, T. D., & Brodsky, J. L. (2018) · PubMed Central · doi:10.1534/genetics.118.301026 · Cited by 19
CK19 stabilizes CFTR at the cell surface by limiting its endocytic pathway degradation
Hou, X., Wu, Q., Rajagopalan, C., Zhang, C., Bouhamdan, M., Wei, H., Chen, X., Zaman, K., Li, C., Sun, X., et al. (2019) · The FASEB Journal · doi:10.1096/fj.201901050r · Cited by 18
Self-citationKrainer, G., Schenkel, M., Hartmann, A. J., Ravamehr-Lake, D., Deber, C. M., & Schlierf, M. (2019) · Journal of Biological Chemistry · doi:10.1074/jbc.ac119.011360 · Cited by 19
Strub, M. D., & McCray, P. B. (2020) · Genes · doi:10.3390/genes11050546 · Cited by 19
Correctors modify the bicarbonate permeability of F508del-CFTR
Fiore, M., Picco, C., & Morán, Ó. (2020) · Scientific Reports · doi:10.1038/s41598-020-65287-4 · Cited by 19
Donaldson, S. H., Laube, B. L., Mogayzel, P. J., Corcoran, T. E., Pilewski, J. M., Ceppe, A., Wu, J., Bhambhvani, P. G., Ratjen, F. A., Sagel, S. D., et al. (2021) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2021.05.004 · Cited by 19
Another Beginning for Cystic Fibrosis Therapy
Davis, P. B. (2015) · New England Journal of Medicine · doi:10.1056/nejme1504059 · Cited by 21
van Mourik, P., Heida-Michel, S., Vonk, A. M., Beekman, J. M., van der Ent, C. K., DeKeyser, H., Lammertyn, E. J., Koningsbruggen-Rietschel, S. V., Naehrlich, L., Pool, J., et al. (2020) · Translational Medicine Communications · doi:10.1186/s41231-020-00060-3 · Cited by 19
Giallongo, A., Parisi, G. F., Papale, M. R., Manti, S., Mulè, E., Aloisio, D., Terlizzi, V., Rotolo, N., & Leonardi, S. (2023) · Genes · doi:10.3390/genes14020449 · Cited by 20
The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?
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Brusa, I., Sondo, E., Pesce, E., Tomati, V., Gioia, D., Falchi, F., Balboni, B., Martínez, J. A. O., Veronesi, M., Romeo, E., et al. (2023) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.3c00608 · Cited by 19
Taniguchi, S., Ono, Y., Doi, Y., Taniguchi, S., Matsuura, Y., Iwasaki, A., Hirata, N., Fukuda, R., Inoue, K., Yamaguchi, M., et al. (2023) · Biochemical Pharmacology · doi:10.1016/j.bcp.2023.115730 · Cited by 19
Reduced PDZ Interactions of Rescued ΔF508CFTR Increases Its Cell Surface Mobility
Valentine, C. D., Lukács, G. L., Verkman, A. S., & Haggie, P. M. (2012) · Journal of Biological Chemistry · doi:10.1074/jbc.m112.421172 · Cited by 20
Ettorre, M., Verzè, G., Caldrer, S., Johansson, J., Calcaterra, E., Assael, B. M., Melotti, P., Sorio, C., & Buffelli, M. (2014) · Biochimica et Biophysica Acta (BBA) - General Subjects · doi:10.1016/j.bbagen.2014.07.010 · Cited by 18
Pharmacological Chaperones: Beyond Conformational Disorders
Leidenheimer, N. J. (2017) · Handbook of experimental pharmacology · doi:10.1007/164_2017_68 · Cited by 19
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Ferrera, L., Baroni, D., & Morán, Ó. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.01.012 · Cited by 18
Re-imagining cystic fibrosis care: next generation thinking
Rang, C., Keating, D., Wilson, J. W., & Kotsimbos, T. C. (2020) · European Respiratory Journal · doi:10.1183/13993003.02443-2019 · Cited by 18
The dual phosphodiesterase 3/4 inhibitor RPL554 stimulates rare class III and IV CFTR mutants
Turner, M. J., Luo, Y., Thomas, D. Y., & Hanrahan, J. W. (2020) · American Journal of Physiology-Lung Cellular and Molecular Physiology · doi:10.1152/ajplung.00285.2019 · Cited by 18
McKone, E. F., DiMango, E., Sutharsan, S., Barto, T. L., Campbell, D., Ahluwalia, N., Higgins, M., Owen, C. A., & Tullis, E. D. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.11.003 · Cited by 19
Cystic fibrosis transmembrane conductance regulator modulators for cystic fibrosis: a new dawn?
Edmondson, C. L., Course, C. W., & Doull, I. J. M. (2021) · Archives of Disease in Childhood · doi:10.1136/archdischild-2020-320680 · Cited by 19
Molecular Dynamics and Theratyping in Airway and Gut Organoids Reveal R352Q-CFTR Conductance Defect
Wong, S. L., Awatade, N. T., Astore, M. A., Allan, K. M., Carnell, M. J., Šlapetová, I., Chen, P., Setiadi, J., Pandzic, E., Fawcett, L. K., et al. (2022) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2021-0337oc · Cited by 15
Merk, D., & Schubert‐Zsilavecz, M. (2013) · Expert Opinion on Drug Discovery · doi:10.1517/17460441.2013.788495 · Cited by 19
Tissue-specific control of CFTR endocytosis by Dab2
Madden, D. R., & Swiatecka‐Urban, A. (2012) · Communicative & Integrative Biology · doi:10.4161/cib.21375 · Cited by 18
Langron, E., Simone, M. I., Delalande, C., Reymond, J., Selwood, D. L., & Vergani, P. (2017) · British Journal of Pharmacology · doi:10.1111/bph.13715 · Cited by 15
Noël, S., Panin, N., Beka, M., Dhooghe, B., Huaux, F., & Leal, T. (2017) · Clinical Science · doi:10.1042/cs20160749 · Cited by 18
Doonan, L. M., Guerriero, C. J., Preston, G. M., Buck, T. M., Khazanov, N., Fisher, E. A. A., Senderowitz, H., & Brodsky, J. L. (2019) · Protein Science · doi:10.1002/pro.3636 · Cited by 18
A novel fluorescent sensor for measurement of CFTR function by flow cytometry
Vijftigschild, L. A. W., van der Ent, C. K., & Beekman, J. M. (2013) · Cytometry Part A · doi:10.1002/cyto.a.22275 · Cited by 18
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Mancini, G., Loberto, N., Olioso, D., Dechecchi, M. C., Cabrini, G., Mauri, L., Bassi, R., Schiumarini, D., Chiricozzi, E., Lippi, G., et al. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21124486 · Cited by 17
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Renda, M., Barreca, M., Borrelli, A., Spanò, V., Montalbano, A., Raimondi, M. V., Bivacqua, R., Musante, I., Scudieri, P., Guidone, D., et al. (2023) · Scientific Reports · doi:10.1038/s41598-023-34440-0 · Cited by 18
Cao, L., Wu, Y., Gong, Y., & Zhou, Q. (2024) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2023.116120 · Cited by 18
Bomberger, J. M., Coutermarsh, B., Barnaby, R., Sato, J. D., Chapline, M. C., & Stanton, B. A. (2014) · PLoS ONE · doi:10.1371/journal.pone.0089599 · Cited by 17
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Cabrini, G. (2018) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-018-0372-6 · Cited by 17
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Matthes, E., Goepp, J., Martini, C., Shan, J., Liao, J., Thomas, D. Y., & Hanrahan, J. W. (2018) · Frontiers in Pharmacology · doi:10.3389/fphar.2018.01490 · Cited by 17
Concilli, M., Petruzzelli, R., Parisi, S., Catalano, F., Sirci, F., Napolitano, F., Renda, M., Galietta, L. J. V., di Bernardo, D., & Polishchuk, R. (2020) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2006648117 · Cited by 17
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Hwang, T., Braakman, I., van der Sluijs, P., & Callebaut, I. (2022) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2022.09.010 · Cited by 16
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Barry, P. J., & Jones, A. M. (2015) · Drugs · doi:10.1007/s40265-015-0424-8 · Cited by 17
Elbahnsi, A., Dudás, B., Callebaut, I., Hinzpeter, A., & Miteva, M. A. (2024) · Pharmaceuticals · doi:10.3390/ph17121602 · Cited by 19
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Tümmler, B., Pallenberg, S. T., Dittrich, A., Graeber, S. Y., Naehrlich, L., Sommerburg, O., & Mall, M. A. (2025) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-025-00194-0 · Cited by 20
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Ong, T., & Ramsey, B. W. (2013) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0b013e328365ab5f · Cited by 16
Fajac, I., & Burgel, P. (2023) · La Presse Médicale · doi:10.1016/j.lpm.2023.104169 · Cited by 16
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Ayca, E., Altintel, B., Levanon, N. L., Ben‐Tal, N., Haliloǧlu, T., & Lewinson, O. (2023) · eLife · doi:10.7554/elife.88659.3 · Cited by 17
Ferreira, F. C., Buarque, C. D., & Lopes‐Pacheco, M. (2024) · Molecules · doi:10.3390/molecules29040821 · Cited by 17
Kolski‐Andreaco, A., Taiclet, S. N., Myerburg, M. M., Sembrat, J., Bridges, R. J., Straub, A. C., Wills, Z. P., Butterworth, M. B., & Devor, D. C. (2024) · Journal of Clinical Investigation · doi:10.1172/jci176328 · Cited by 16
Baroni, D. (2025) · Current Issues in Molecular Biology · doi:10.3390/cimb47020119 · Cited by 17
Metadata
- Authors
- Van Goor, F., Hadida, S., Grootenhuis, P. D. J., Burton, B., Stack, J. H., Straley, K. S., Decker, C. J., Miller, M., McCartney, J., Olson, E. R., Wine, J. J., Frizzell, R. A., Ashlock, M., & Negulescu, P. A.
- Affiliations
- Vertex Pharmaceuticals, San Diego, CA 92121;
- Vertex Pharmaceuticals (United States) (US)
- Stanford University (US)
- Department of Cell Biology and Physiology, University of Pittsburgh School of Medicine, Pittsburgh, PA 15261; and
- Cystic Fibrosis Foundation (US)
- Corresponding author
- Negulescu, Paul A.
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2011-10-05
- Volume
- 108
- Issue
- 46
- Pages
- 18843-18848
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC3219147
- OpenAlex
- W2071407356
- Cited by
- 1,088
- References
- 44
- Citations by year
- 2026: 24 · 2025: 48 · 2024: 48 · 2023: 57 · 2022: 64 · 2021: 83 · 2020: 98 · 2019: 95 · 2018: 101 · 2017: 103 · 2016: 77 · 2015: 85 · 2014: 84 · 2013: 61 · 2012: 58
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Cystic Fibrosis Foundation Therapeutics
- Topics
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Ion Transport and Channel Regulation
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Keywords
- cystic fibrosis, F508del-CFTR, CFTR correctors, lumacaftor, endoplasmic reticulum retention, chloride secretion, bronchial epithelial cells, CFTR mutations
- MeSH terms
- Aminopyridines, Bronchi, Cell Line, Cells, Cultured, Chemistry, Pharmaceutical, Chlorides, Cystic Fibrosis, Drug Evaluation, Preclinical, Epithelial Cells, Homozygote, Humans, Lung, Models, Genetic, Mutation, Drug Design, Cystic Fibrosis Transmembrane Conductance Regulator, Benzodioxoles, In Vitro Techniques
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