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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 601–700 of 1,000
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Anjos, S. M., Robert, R., Waller, D., Zhang, D. L., Balghi, H., Sampson, H. M., Ciciriello, F., Lesimple, P., Carlile, G. W., Goepp, J., et al. (2012) · Frontiers in Pharmacology · doi:10.3389/fphar.2012.00165 · Cited by 16
McShane, A. J., Bajrami, B., Ramos, A. A., Diego, P. A., Farrokhi, V., Coutermarsh, B., Stanton, B. A., Jensen, T., Riordan, J. R., Wetmore, D. R., et al. (2014) · Journal of Proteome Research · doi:10.1021/pr5006795 · Cited by 16
Banerjee, M., Arutyunov, D., Brandwein, D., Janetzki‐Flatt, C., Kolski, H., Hume, S. L., Leonard, N., Watt, J., Lacson, A. G., Baradi, M., et al. (2019) · Human Mutation · doi:10.1002/humu.23828 · Cited by 16
Brindani, N., Gianotti, A., Giovani, S., Giacomina, F., Fruscia, P. D., Sorana, F., Bertozzi, S. M., Ottonello, G., Goldoni, L., Penna, I., et al. (2020) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.0c01050 · Cited by 16
Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E–CFTR mutation
Berkers, G., van der Meer, R., Heijerman, H. G. M., Beekman, J. M., Boj, S. F., Vries, R. G. J., van Mourik, P., Doyle, J. R., Audhya, P., Yuan, Z. (., et al. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.11.007 · Cited by 17
Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis
Conti, J., Sorio, C., & Melotti, P. (2022) · Children · doi:10.3390/children10010004 · Cited by 18
Bridges, R. J., & Bradbury, N. A. (2018) · Handbook of experimental pharmacology · doi:10.1007/164_2018_103 · Cited by 16
Rescue of CFTR NBD2 mutants N1303K and S1235R is influenced by the functioning of the autophagosome
Liu, Q., Sabirzhanova, I. B., Yanda, M. K., Bergbower, E. A. S., Boinot, C., Guggino, W. B., & Cebotaru, L. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.05.016 · Cited by 16
Yeh, J., Yu, Y., & Hwang, T. (2018) · The Journal of Physiology · doi:10.1113/jp277042 · Cited by 16
Froux, L., Elbahnsi, A., Boucherle, B., Billet, A., Baatallah, N., Hoffmann, B., Alliot, J., Zelli, R., Zeinyeh, W., Haudecoeur, R., et al. (2020) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2020.112116 · Cited by 16
An unfolding story: Small molecules remedy misfolded monoamine transporters
Kasture, A. S., Stockner, T., Freissmuth, M., & Sučić, S. (2017) · The International Journal of Biochemistry & Cell Biology · doi:10.1016/j.biocel.2017.09.004 · Cited by 16
Mechanism of dual pharmacological correction and potentiation of human CFTRPreprint
Wang, C., Yang, Z. W., Loughlin, B. J., Xu, H., Veit, G., Vorobiev, S. M., Clarke, O. B., Jiang, F., Li, Y., Singh, S., et al. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.10.10.510913 · Cited by 17
Nguyen, J., Huff, R. D., Cao, Q., Tiessen, N., Carlsten, C., & Hirota, J. A. (2021) · Toxicology in Vitro · doi:10.1016/j.tiv.2021.105253 · Cited by 15
Discovery and Development of CFTR Modulators for the Treatment of Cystic Fibrosis
Wang, X., Tse, C., & Singh, A. K. (2025) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.4c02547 · Cited by 15
Simple image-based no-wash method for quantitative detection of surface expressed CFTR
Larsen, M. B., Hu, J., Frizzell, R. A., & Watkins, S. C. (2015) · Methods · doi:10.1016/j.ymeth.2015.09.006 · Cited by 15
Self-citationTherapeutic Targeting of Misfolding and Conformational Change in α 1 -Antitrypsin Deficiency
Nyon, M. P., & Gooptu, B. (2014) · Future Medicinal Chemistry · doi:10.4155/fmc.14.58 · Cited by 15
Wang, Y., Cai, Z., Gosling, M., & Sheppard, D. N. (2018) · American Journal of Physiology-Lung Cellular and Molecular Physiology · doi:10.1152/ajplung.00235.2018 · Cited by 15
Tucker, T. A., Fortenberry, J. A., Zsembery, Á., Schwiebert, L. M., & Schwiebert, E. M. (2012) · BMC Physiology · doi:10.1186/1472-6793-12-12 · Cited by 15
Pesce, E., Gorrieri, G., Sirci, F., Napolitano, F., Carrella, D., Caci, E., Tomati, V., Zegarra‐Moran, O., di Bernardo, D., & Galietta, L. J. V. (2016) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2016.02.009 · Cited by 15
A Pharmacological Chaperone Therapy for Acute Intermittent Porphyria
Bustad, H. J., Toska, K., Schmitt, C., Vorland, M., Skjærven, L., Kallio, J. P., Simonin, S., Lettéron, P., Underhaug, J., Sandberg, S., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.11.010 · Cited by 15
Yanda, M. K., & Cebotaru, L. (2021) · The FASEB Journal · doi:10.1096/fj.202101315r · Cited by 14
Cystic Fibrosis: therapies targeting specific gene defects
Thursfield, R., & Davies, J. C. (2012) · Paediatric Respiratory Reviews · doi:10.1016/j.prrv.2012.04.003 · Cited by 14
Long-term effectiveness of dual CFTR modulator treatment of cystic fibrosis
Muilwijk, D., Ommen, D. D. Z.-V., Gulmans, V. A. M., Eijkemans, M. J. C., van der Ent, C. K., Dutch Cystic Fibrosis Registry (NCFR) Steering Group:, Altenburg, J., Terheggen‐Lagro, S. W. J., Heijerman, H. G. M., Groot, K. M. D. W., et al. (2022) · ERJ Open Research · doi:10.1183/23120541.00204-2022 · Cited by 14
Redefining Hypo- and Hyper-Responding Phenotypes of CFTR Mutants for Understanding and Therapy
Hillenaar, T., Beekman, J. M., van der Sluijs, P., & Braakman, I. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232315170 · Cited by 14
Function, pharmacological correction and maturation of new Indian CFTR gene mutations
Sharma, H., Souchet, M. J., Callebaut, I., Prasad, R., & Becq, F. (2014) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2014.06.008 · Cited by 14
Dilokthornsakul, P., Patidar, M., & Campbell, J. D. (2017) · Value in Health · doi:10.1016/j.jval.2017.06.014 · Cited by 14
Harrison, P. T., Hoppe, N., & Martin, U. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.11.018 · Cited by 14
Shimshilashvili, L., Aharon, S., Moe, O. W., & Ohana, E. (2020) · Frontiers in Pharmacology · doi:10.3389/fphar.2020.00405 · Cited by 14
Wong, S. L., Awatade, N. T., Astore, M. A., Allan, K. M., Carnell, M. J., Šlapetová, I., Chen, P., Capraro, A., Fawcett, L. K., Whan, R. M., et al. (2021) · iScience · doi:10.1016/j.isci.2021.103710 · Cited by 14
Membrane proteins enter the fold
Marx, D. C., & Fleming, K. G. (2021) · Current Opinion in Structural Biology · doi:10.1016/j.sbi.2021.03.006 · Cited by 13
Chatterjee, P., Moss, C. T., Omar, S., Dhillon, E., Borges, C. H., Tang, A. C., Stevens, D. A., & Hsu, J. L. (2024) · Journal of Fungi · doi:10.3390/jof10090656 · Cited by 13
Insulin-Like Growth Factor 1 (IGF-1) Enhances the Protein Expression of CFTR
Lee, H. W., Cheng, J., Kovbasnjuk, O. N., Donowitz, M., & Guggino, W. B. (2013) · PLoS ONE · doi:10.1371/journal.pone.0059992 · Cited by 13
CFTR Protein Repair Therapy in Cystic Fibrosis
Quintana‐Gallego, E., Delgado‐Pecellín, I., & Acuña, C. C. (2014) · Archivos de Bronconeumología · doi:10.1016/j.arbr.2014.03.002 · Cited by 13
Bali, V., Lazrak, A., Guroji, P., Matalon, S., & Bebok, Z. M. (2016) · PLoS ONE · doi:10.1371/journal.pone.0155882 · Cited by 13
Righetti, G., Casale, M., Liessi, N., Tasso, B., Salis, A., Tonelli, M., Millo, E., Pedemonte, N., Fossa, P., & Cichero, E. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21218084 · Cited by 13
Goeckeler-Fried, J. L., Denny, R. A., Joshi, D., Hill, C., Larsen, M. B., Chiang, A., Frizzell, R. A., Wipf, P., Sorscher, E. J., & Brodsky, J. L. (2021) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2021.128243 · Cited by 13
Self-citationTerlizzi, V., Centrone, C., Ferrari, B., Castellani, C., Gunawardena, T. N. A., Taccetti, G., & Laselva, O. (2022) · Journal of Personalized Medicine · doi:10.3390/jpm12091421 · Cited by 13
Chu, C., King, J., Berrini, M., Rumley, A. C., Apaja, P. M., Lukács, G. L., Alexander, R. T., & Cordat, E. (2014) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00310.2013 · Cited by 13
Tratamientos reparadores de la proteína CFTR en la fibrosis quística
Quintana‐Gallego, E., Delgado‐Pecellín, I., & Acuña, C. C. (2013) · Archivos de Bronconeumología · doi:10.1016/j.arbres.2013.07.013 · Cited by 13
Markovetz, M. R., Corcoran, T. E., Locke, L. W., Myerburg, M. M., Pilewski, J. M., & Parker, R. S. (2014) · PLoS ONE · doi:10.1371/journal.pone.0111972 · Cited by 13
Csanády, L., & Törőcsik, B. (2014) · The Journal of General Physiology · doi:10.1085/jgp.201411246 · Cited by 13
Ivacaftor for the treatment of cystic fibrosis in children under six years of age
Aoyama, B. C., & Mogayzel, P. J. (2020) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2020.1741352 · Cited by 12
Parodi, A. C. L., Righetti, G., Pesce, E., Salis, A., Tasso, B., Urbinati, C., Tomati, V., Damonte, G., Rusnati, M., Pedemonte, N., et al. (2020) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2020.112833 · Cited by 13
Bratcher, P. E., Yadav, S., Shaughnessy, C. A., Thornell, I. M., & Zeitlin, P. L. (2020) · Physiological Reports · doi:10.14814/phy2.14603 · Cited by 13
Shioi, R., Karaki, F., YOSHIOKA, H., Noguchi‐Yachide, T., Ishikawa, M., Dodo, K., Hashimoto, Y., Sodeoka, M., & Ohgane, K. (2020) · PLoS ONE · doi:10.1371/journal.pone.0243746 · Cited by 13
Phenylhydrazones as Correctors of a Mutant Cystic Fibrosis Transmembrane Conductance Regulator
Nieddu, E., Pollarolo, B., Mazzei, M. T., Anzaldi, M., Schenone, S., Pedemonte, N., Galietta, L. J. V., & Mazzei, M. (2015) · Archiv der Pharmazie · doi:10.1002/ardp.201500352 · Cited by 13
Long-term management of patients with end-stage lung diseases
Briganti, D. F., & D’Ovidio, F. (2017) · Best practice & research. Clinical anaesthesiology · doi:10.1016/j.bpa.2017.07.007 · Cited by 18
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Alméciga-Díaz, C. J., Pimentel-Vera, L. N., Caro, A., Mosquera, Á., Moreno, C. N. A., Rojas, J. P. M., & Díaz-Tribaldos, D. C. (2020) · Preprints.org · doi:10.20944/preprints202004.0146.v1 · Cited by 13
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Bihler, H. J., Sivachenko, A., Millen, L., Bhatt, P., Thakerar, A., Chin, J., Bailey, V., Musisi, I., LaPan, A., Allaire, N., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.07.07.548159 · Cited by 13
Centko, R. M., Carlile, G. W., Barne, I., Patrick, B. O., Blagojević, P. D., Thomas, D. Y., & Andersen, R. J. (2020) · ACS Omega · doi:10.1021/acsomega.0c02467 · Cited by 12
Small molecule modulation of protein polymerization
Fischer, E. S., & Jones, L. H. (2022) · Chemical Society Reviews · doi:10.1039/d2cs00070a · Cited by 16
Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states
Riepe, C., Wąchalska, M., Deol, K. K., Amaya, A. K., Porteus, M. H., Olzmann, J. A., & Kopito, R. R. (2023) · Molecular Biology of the Cell · doi:10.1091/mbc.e23-08-0336 · Cited by 12
Thakur, S., Ankita, A., Dash, S., Verma, R., Kaur, C., Kumar, R., Mazumder, A., & Singh, G. (2023) · Cell Biochemistry and Biophysics · doi:10.1007/s12013-023-01200-w · Cited by 12
A uniquely efficacious type of CFTR corrector with complementary mode of action
Marchesin, V., Monnier, L., Blattmann, P., Chevillard, F., Kuntz, C., Forny, C., Kamper, J., Studer, R. O., Bossu, A., Ertel, E. A., et al. (2024) · Science Advances · doi:10.1126/sciadv.adk1814 · Cited by 12
HERC3 facilitates ERAD of select membrane proteins by recognizing membrane-spanning domains
Kamada, Y., Ohnishi, Y., Nakashima, C., Fujii, A., Terakawa, M., Hamano, I., Nakayamada, U., Katoh, S., Hirata, N., Tateishi, H., et al. (2024) · The Journal of Cell Biology · doi:10.1083/jcb.202308003 · Cited by 14
Estácio, S. G., Martiniano, H., & Faísca, P. F. N. (2016) · Molecular BioSystems · doi:10.1039/c6mb00193a · Cited by 11
Llinares, J., Cantereau, A., Froux, L., & Becq, F. (2020) · PLoS ONE · doi:10.1371/journal.pone.0233439 · Cited by 12
New Therapeutic Approaches in Cystic Fibrosis
Fakıoğlu, D. M., & Altun, B. (2020) · Turkish Journal of Pharmaceutical Sciences · doi:10.4274/tjps.galenos.2020.76401 · Cited by 12
Bitam, S., Elbahnsi, A., Creste, G., Pranke, I. M., Chevalier, B., Berhal, F., Hoffmann, B., Servel, N., Baatalah, N., Tondelier, D., et al. (2021) · Scientific Reports · doi:10.1038/s41598-021-83240-x · Cited by 12
DNA-modularized construction of bivalent ligands precisely regulates receptor binding and activation
Zhao, N., Wu, W., Wang, Y., Song, K., Chen, G., Chen, Y., Wang, R., Xu, J., Cui, K., Chen, H., et al. (2023) · Chem · doi:10.1016/j.chempr.2022.12.002 · Cited by 12
Ramachandran, S. S., Osterhaus, S. R., Karp, P. H., Welsh, M. J., & McCray, P. B. (2014) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2014-0007oc · Cited by 12
Norman, P. (2014) · Expert Opinion on Therapeutic Patents · doi:10.1517/13543776.2014.876412 · Cited by 12
Gaining the Upper Hand on Pulmonary Drug Delivery
Tyrrell, R. T. J. (2014) · Journal of Pharmacovigilance · doi:10.4172/2329-6887.1000118 · Cited by 11
Yin, D., Wang, W., Peng, Y., Ge, Z., Cheng, T., Wang, X., & Li, R. (2014) · RSC Advances · doi:10.1039/c4ra13796e · Cited by 12
Pollock, N. L., Satriano, L., Zegarra‐Moran, O., Ford, R. C., & Morán, Ó. (2016) · Journal of Structural Biology · doi:10.1016/j.jsb.2016.02.004 · Cited by 12
Wang, C. C., Aleksandrov, A. A., Yang, Z. W., Forouhar, F., Proctor, E. A., Kota, P. B., An, J., Kaplan, A., Khazanov, N., Boël, G., et al. (2018) · Journal of Biological Chemistry · doi:10.1074/jbc.ra117.000819 · Cited by 12
Cottrill, K. A., Peterson, R. J., Lewallen, C. F., Koval, M. H., Bridges, R. J., & McCarty, N. A. (2021) · Physiological Reports · doi:10.14814/phy2.14928 · Cited by 12
Wei, W., Zheng, C., Zhu, M., Zhu, X., Yang, R., Misra, S., & Zhang, B. (2017) · Scientific Reports · doi:10.1038/srep45033 · Cited by 12
Germline genetic variants with implications for disease risk and therapeutic outcomes
Pasternak, A. L., Ward, K. M., Luzum, J. A., Ellingrod, V. L., & Hertz, D. L. (2017) · Physiological Genomics · doi:10.1152/physiolgenomics.00035.2017 · Cited by 12
Bacalhau, M., Camargo, M., & Lopes‐Pacheco, M. (2024) · Journal of Personalized Medicine · doi:10.3390/jpm14010093 · Cited by 11
Bali, V., Lazrak, A., Guroji, P., Fu, L., Matalon, S., & Bebok, Z. M. (2015) · The FASEB Journal · doi:10.1096/fj.15-273714 · Cited by 12
Three-Dimensional Airway Spheroids and Organoids for Cystic Fibrosis Research
Laselva, O., & Conese, M. (2021) · Journal of Respiration · doi:10.3390/jor1040022 · Cited by 12
McElvaney, O. J., Heltshe, S. L., Odem‐Davis, K., West, N. E., Sanders, D. B., Fogarty, B., VanDevanter, D. R., Flume, P. A., & Goss, C. H. (2023) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2023.06.012 · Cited by 11
Towards personalized agriculture: what chemical genomics can bring to plant biotechnology
Stokes, M. E., & McCourt, P. (2014) · Frontiers in Plant Science · doi:10.3389/fpls.2014.00344 · Cited by 12
Cai, Z., Li, H., Chen, J.-H., & Sheppard, D. N. (2013) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00052.2013 · Cited by 11
Molina, S. A., & Hunt, W. R. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-803809-3.00012-9 · Cited by 13
Cystic Fibrosis: Proteostatic correctors of CFTR trafficking and alternative therapeutic targets.
Hanrahan, J. W., Sato, Y., Carlile, G. W., Jansen, G., Young, J. C., & Thomas, D. Y. (2019) · Expert Opinion on Therapeutic Targets · doi:10.1080/14728222.2019.1628948 · Cited by 10
Froux, L., Coraux, C., Sage, É., & Becq, F. (2019) · Scientific Reports · doi:10.1038/s41598-019-50066-7 · Cited by 11
Validation of nasospheroids to assay CFTR functionality and modulator responses in cystic fibrosis
Calucho, M., Gärtner, S., Barranco, P., Fernández‐Álvarez, P., Pérez, R. G., & Tizzano, E. F. (2021) · Scientific Reports · doi:10.1038/s41598-021-94798-x · Cited by 12
Efficacy of CFTR modulators in clinical practice (6-month follow-up)
Kondratyeva, E., Odinaeva, N. D., Zhekaite, E. K., Pasnova, E. V., Fatkhullina, I. R., Maksimycheva, T., Sherman, V. D., & Kutsev, S. I. (2023) · PULMONOLOGIYA · doi:10.18093/0869-0189-2023-33-2-189-197 · Cited by 10
Restoring CFTR function with Orkambi decreases the severity of alcohol‐induced acute pancreatitis
Venglovecz, V., Grassalkovich, A., Tóth, E., Èbert, A., Gál, E., Korsós, M. M., Maléth, J., Rakonczay, Z., Galla, Z., Monostori, P. B., et al. (2024) · The Journal of Physiology · doi:10.1113/jp287289 · Cited by 10
Gechijian, L., Muncipinto, G., Rettenmaier, T. J., Labenski, M., Rusu, V., Rosskamp, L., Conway, L., van Kalken, D., Gross, L., Iantosca, G., et al. (2024) · ACS Chemical Biology · doi:10.1021/acschembio.4c00571 · Cited by 10
Yang, H. Y., & Ma, T. (2015) · Expert Opinion on Therapeutic Patents · doi:10.1517/13543776.2015.1045878 · Cited by 10
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Carlile, G. W., Yang, Q., Matthes, E., Liao, J., Birault, V., Sneddon, H. F., Poole, D. L., Hall, C. J., Hanrahan, J. W., & Thomas, D. Y. (2022) · Scientific Reports · doi:10.1038/s41598-022-08661-8 · Cited by 10
Benoit, I., Burty-Valin, E., & Radman, M. (2023) · Clinical Cosmetic and Investigational Dermatology · doi:10.2147/ccid.s397751 · Cited by 11
Laval, F., Coppin, G., Twizere, J., & Vidal, M. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24119179 · Cited by 10
Carbone, A., Vitullo, P., Gioia, S. D., & Conese, M. (2023) · Genes · doi:10.3390/genes14101966 · Cited by 10
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Farinha, C. M., Miller, E. A., & McCarty, N. A. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.08.014 · Cited by 10
Bajko, J., Duguid, M., Altmann, S., Hurlbut, G. D., & Kaczmarek, J. S. (2020) · FASEB BioAdvances · doi:10.1096/fba.2020-00012 · Cited by 10
Righetti, G., Casale, M., Tonelli, M., Liessi, N., Fossa, P., Pedemonte, N., Millo, E., & Cichero, E. (2020) · Pharmaceuticals · doi:10.3390/ph13120445 · Cited by 10
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Nguyen, J., Bianca, M., Huff, R. D., Tiessen, N., Inman, M. D., & Hirota, J. A. (2021) · Scientific Reports · doi:10.1038/s41598-020-79555-w · Cited by 10
Vinhoven, L., Stanke, F., Hafkemeyer, S., & Nietert, M. M. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22147590 · Cited by 10
Martina, M. G., Sannio, F., Crespan, E., Pavone, M., Simoncini, A., Barbieri, F., Perini, C., Pesce, E., Maga, G., Pedemonte, N., et al. (2022) · ChemMedChem · doi:10.1002/cmdc.202200277 · Cited by 10
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Yeh, H., Sutcliffe, K. J., Sheppard, D. N., & Hwang, T. (2022) · Handbook of experimental pharmacology · doi:10.1007/164_2022_597 · Cited by 10
Zacarias, S., Batista, M. S. P., Ramalho, S. S., Victor, B. L., & Farinha, C. M. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24043211 · Cited by 10
Yarlagadda, S., Zhang, W., Penmatsa, H., Ren, A., Arora, K., Naren, A. P., Khan, F. A. I., Donnellan, C. A., Srinivasan, S., Stokes, D. C., et al. (2012) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/ajrccm.186.7.694 · Cited by 10
Adnan, H., Zhang, Z., Park, H.-J., Tailor, C. S., Che, C., Kamani, M., Spitalny, G. L., Binnington, B., & Lingwood, C. A. (2016) · PLoS ONE · doi:10.1371/journal.pone.0166948 · Cited by 10
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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