{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T14:51:44Z","timestamp":1787496704755,"version":"build-2736575974"},"reference-count":72,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T00:00:00Z","timestamp":1707264000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["LOPES23G0"],"award-info":[{"award-number":["LOPES23G0"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["LOPES21I0"],"award-info":[{"award-number":["LOPES21I0"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001871","name":"Foundation for Science and Technology","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001871","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["European Journal of Pharmacology"],"published-print":{"date-parts":[[2024,3]]},"DOI":"10.1016\/j.ejphar.2024.176390","type":"journal-article","created":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T22:38:58Z","timestamp":1707345538000},"page":"176390","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":10,"special_numbering":"C","title":["PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR"],"prefix":"10.1016","volume":"967","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7335-6997","authenticated-orcid":false,"given":"Filipa C.","family":"Ferreira","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margarida D.","family":"Amaral","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8857-3329","authenticated-orcid":false,"given":"Mafalda","family":"Bacalhau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7444-9359","authenticated-orcid":false,"given":"Miqu\u00e9ias","family":"Lopes-Pacheco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ejphar.2024.176390_bib1","doi-asserted-by":"crossref","first-page":"742","DOI":"10.1002\/humu.23741","article-title":"Two CFTR mutations within codon 970 differently impact on the chloride channel functionality","volume":"40","author":"Amato","year":"2019","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ejphar.2024.176390_bib2","doi-asserted-by":"crossref","first-page":"7813","DOI":"10.1007\/s00018-021-03994-5","article-title":"Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR","volume":"78","author":"Baatallah","year":"2021","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.ejphar.2024.176390_bib71","doi-asserted-by":"crossref","first-page":"93","DOI":"10.3390\/jpm14010093","article-title":"Laboratory tools to predict CFTR modulator therapy effectiveness and to monitor disease severity in cystic fibrosis","volume":"14","author":"Bacalhau","year":"2024","journal-title":"J. Pers. Med."},{"key":"10.1016\/j.ejphar.2024.176390_bib3","doi-asserted-by":"crossref","first-page":"410","DOI":"10.3390\/ph16030410","article-title":"Elexacaftor-tezacaftor-ivacaftor: a life-changing triple combination of CFTR modulator drugs for cystic fibrosis","volume":"16","author":"Bacalhau","year":"2023","journal-title":"Pharmaceuticals"},{"key":"10.1016\/j.ejphar.2024.176390_bib4","doi-asserted-by":"crossref","DOI":"10.1016\/j.ejphar.2022.175396","article-title":"Identification of novel F508del-CFTR traffic correctors among triazole derivatives","volume":"938","author":"Bacalhau","year":"2023","journal-title":"Eur. J. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib5","doi-asserted-by":"crossref","first-page":"102","DOI":"10.3390\/jpm13010102","article-title":"Additive potentiation of R334W-CFTR function by novel small molecules","volume":"13","author":"Bacalhau","year":"2023","journal-title":"J. Pers. Med."},{"key":"10.1016\/j.ejphar.2024.176390_bib6","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1021\/jm901137j","article-title":"New substructure filters for removal of pan assay interference compounds (PAINS) from screening libraries and for their exclusion in bioassays","volume":"53","author":"Baell","year":"2010","journal-title":"J. Med. Chem."},{"key":"10.1016\/j.ejphar.2024.176390_bib7","doi-asserted-by":"crossref","first-page":"601","DOI":"10.1113\/jphysiol.2005.096669","article-title":"Failure of cAMP agonists to activate rescued \u0394F508 CFTR in CFBE41o- airway epithelial monolayers","volume":"569","author":"Bebok","year":"2005","journal-title":"J. Physiol."},{"key":"10.1016\/j.ejphar.2024.176390_bib8","doi-asserted-by":"crossref","first-page":"516","DOI":"10.1164\/rccm.201507-1281OC","article-title":"Morphometric analysis of explant lungs in cystic fibrosis","volume":"193","author":"Boon","year":"2016","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.ejphar.2024.176390_bib9","doi-asserted-by":"crossref","first-page":"9038","DOI":"10.1038\/srep09038","article-title":"Protein traffic Disorders : an effective high-throughput fluorescence microscopy Pipeline for drug Discovery","volume":"12","author":"Botelho","year":"2015","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ejphar.2024.176390_bib10","doi-asserted-by":"crossref","first-page":"5262","DOI":"10.3390\/ijms22105262","article-title":"Partial rescue of f508del-cftr stability and trafficking defects by double corrector treatment","volume":"22","author":"Capurro","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ejphar.2024.176390_bib11","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1016\/S1097-2765(00)80196-3","article-title":"Removal of multiple arginine-framed trafficking signals overcomes misprocessing of \u0394F508 CFTR present in most patients with cystic fibrosis","volume":"4","author":"Chang","year":"1999","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib12","first-page":"129","article-title":"Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis","volume":"83","author":"Cheng","year":"1990","journal-title":"Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib13","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1186\/s13023-022-02350-5","article-title":"The impact of FDA and EMA regulatory decision - making process on the access to CFTR modulators for the treatment of cystic fibrosis","volume":"17","author":"Costa","year":"2022","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ejphar.2024.176390_bib14","doi-asserted-by":"crossref","first-page":"707","DOI":"10.1152\/physrev.00007.2018","article-title":"Structure, gating, and regulation of the CFTR anion channel","volume":"99","author":"Csan\u00e1dy","year":"2019","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.ejphar.2024.176390_bib15","doi-asserted-by":"crossref","DOI":"10.1038\/srep42717","article-title":"SwissADME: a free web tool to evaluate pharmacokinetics, drug-likeness and medicinal chemistry friendliness of small molecules","volume":"7","author":"Daina","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ejphar.2024.176390_bib16","doi-asserted-by":"crossref","first-page":"526","DOI":"10.1038\/354526a0","article-title":"Altered chloride ion channel kinetics associated with the delta F508 cystic fibrosis mutation","volume":"354","author":"Dalemans","year":"1991","journal-title":"Nature"},{"key":"10.1016\/j.ejphar.2024.176390_bib17","doi-asserted-by":"crossref","first-page":"35896","DOI":"10.1074\/jbc.M205644200","article-title":"Mutations in the nucleotide binding domain 1 signature motif region rescue processing and functional defects of cystic fibrosis transmembrane conductance regulator \u03b4F508","volume":"277","author":"Decarvalho","year":"2002","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ejphar.2024.176390_bib18","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1038\/358761a0","article-title":"Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive","volume":"358","author":"Denning","year":"1992","journal-title":"Nature"},{"key":"10.1016\/j.ejphar.2024.176390_bib19","doi-asserted-by":"crossref","first-page":"943","DOI":"10.1016\/j.chembiol.2013.06.004","article-title":"Revertants, low temperature, and correctors reveal the mechanism of F508del-CFTR rescue by VX-809 and suggest multiple agents for full correction","volume":"20","author":"Farinha","year":"2013","journal-title":"Chem. Biol."},{"key":"10.1016\/j.ejphar.2024.176390_bib20","doi-asserted-by":"crossref","first-page":"S74","DOI":"10.1016\/S1569-1993(23)00410-1","article-title":"P035. Partial correction of F508del-CFTR trafficking and stability defects by the combination of PTI-801 with ABBV-2222 or FDL-169","volume":"22","author":"Ferreira","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib72","doi-asserted-by":"crossref","first-page":"821","DOI":"10.3390\/molecules29040821","article-title":"Organic synthesis and current understanding of the mechanisms of CFTR modulator drugs ivacaftor, tezacaftor, elexacaftor","volume":"29","author":"Ferreira","year":"2024","journal-title":"Molecules"},{"issue":"80-","key":"10.1016\/j.ejphar.2024.176390_bib21","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1126\/science.ade2216","article-title":"Molecular structures reveal synergistic rescue of \u0394508 CFTR by Trikafta modulators","volume":"378","author":"Fiedorczuk","year":"2022","journal-title":"Science"},{"key":"10.1016\/j.ejphar.2024.176390_bib22","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1016\/j.cell.2021.12.009","article-title":"Mechanism of CFTR correction by type I folding correctors","volume":"185","author":"Fiedorczuk","year":"2022","journal-title":"Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib23","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.jcf.2022.01.009","article-title":"Worldwide rates of diagnosis and effective treatment for cystic fibrosis","volume":"21","author":"Guo","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib24","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.jcf.2022.04.007","article-title":"Current prices versus minimum costs of production for CFTR modulators","volume":"21","author":"Guo","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib25","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1096\/fj.12-216119","article-title":"Correctors of F508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein","volume":"27","author":"He","year":"2013","journal-title":"Faseb. J."},{"key":"10.1016\/j.ejphar.2024.176390_bib26","doi-asserted-by":"crossref","first-page":"1940","DOI":"10.1016\/S0140-6736(19)32597-8","article-title":"Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial","volume":"394","author":"Heijerman","year":"2019","journal-title":"Lancet"},{"key":"10.1016\/j.ejphar.2024.176390_bib27","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1124\/mol.117.108373","article-title":"Direct binding of the corrector VX-809 to human CFTR NBD1: evidence of an allosteric coupling between the binding site and the NBD1:CL4 interface s","volume":"92","author":"Hudson","year":"2017","journal-title":"Mol. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib28","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1007\/s00018-022-04671-x","article-title":"ABC-transporter CFTR folds with high fidelity through a modular, stepwise pathway","volume":"80","author":"Im","year":"2023","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.ejphar.2024.176390_bib29","series-title":"Pediatric Pulmonology","first-page":"S148","article-title":"P261. Initial results evaluating third generation CFTR corrector PTI-801 in CF subjects","author":"Jain","year":"2018"},{"key":"10.1016\/j.ejphar.2024.176390_bib30","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1016\/0092-8674(95)90241-4","article-title":"Multiple proteolytic systems, including the proteasome, contribute to CFTR processing","volume":"83","author":"Jensen","year":"1995","journal-title":"Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib31","doi-asserted-by":"crossref","DOI":"10.1183\/13993003.02774-2020","article-title":"Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator","volume":"57","author":"Laselva","year":"2021","journal-title":"Eur. Respir. J."},{"issue":"Suppl. 2","key":"10.1016\/j.ejphar.2024.176390_bib32","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.jcf.2004.05.026","article-title":"Transepithelial electrical measurements with the Ussing chamber","volume":"3","author":"Li","year":"2004","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib33","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1016\/j.ddtec.2004.11.007","article-title":"Lead- and drug-like compounds: the rule-of-five revolution","volume":"1","author":"Lipinski","year":"2004","journal-title":"Drug Discov. Today Technol."},{"key":"10.1016\/j.ejphar.2024.176390_bib34","doi-asserted-by":"crossref","first-page":"6352","DOI":"10.1021\/bi100807h","article-title":"The V510D suppressor mutation stabilizes \u03b4f508-CFTR at the cell surface","volume":"49","author":"Loo","year":"2010","journal-title":"Biochemistry"},{"key":"10.1016\/j.ejphar.2024.176390_bib35","doi-asserted-by":"crossref","first-page":"1662","DOI":"10.3389\/fphar.2019.01662","article-title":"CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine","volume":"10","author":"Lopes-Pacheco","year":"2020","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib36","doi-asserted-by":"crossref","first-page":"136","DOI":"10.3390\/cells11010136","article-title":"Rescue of mutant CFTR trafficking defect by the investigational compound MCG1516A","volume":"11","author":"Lopes-Pacheco","year":"2022","journal-title":"Cells"},{"key":"10.1016\/j.ejphar.2024.176390_bib37","doi-asserted-by":"crossref","first-page":"25636","DOI":"10.1074\/jbc.M115.671925","article-title":"Combination of correctors rescue \u03b4f508-CFTR by reducing its association with Hsp40 and Hsp27","volume":"290","author":"Lopes-Pacheco","year":"2015","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ejphar.2024.176390_bib38","doi-asserted-by":"crossref","first-page":"2194","DOI":"10.1159\/000475578","article-title":"Combination of correctors rescues CFTR transmembrane-domain mutants by mitigating their interactions with proteostasis","volume":"41","author":"Lopes-Pacheco","year":"2017","journal-title":"Cell. Physiol. Biochem."},{"key":"10.1016\/j.ejphar.2024.176390_bib39","doi-asserted-by":"crossref","first-page":"S58","DOI":"10.1016\/S1569-1993(22)00328-9","article-title":"ePS6.01 Characterisation of F508del-CFTR rescue by corrector PTI-801","volume":"21","author":"Lopes-Pacheco","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib40","doi-asserted-by":"crossref","first-page":"897","DOI":"10.1080\/17460441.2021.1912732","article-title":"Discovery of CFTR modulators for the treatment of cystic fibrosis","volume":"16","author":"Lopes-Pacheco","year":"2021","journal-title":"Expet Opin. Drug Discov."},{"key":"10.1016\/j.ejphar.2024.176390_bib41","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1002\/cbic.201500620","article-title":"Correctors rescue CFTR mutations in nucleotide-binding domain 1 (NBD1) by modulating proteostasis","volume":"17","author":"Lopes-Pacheco","year":"2016","journal-title":"Chembiochem"},{"key":"10.1016\/j.ejphar.2024.176390_bib42","doi-asserted-by":"crossref","DOI":"10.1016\/j.bcp.2020.114133","article-title":"Characterization of the mechanism of action of RDR01752, a novel corrector of F508del-CFTR","volume":"180","author":"Lopes-Pacheco","year":"2020","journal-title":"Biochem. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib43","first-page":"599","article-title":"Real-world outcomes and direct care cost before and after elexacaftor\/tezacaftor\/ivacaftor initiation in commercially insured members with cystic fibrosis","volume":"29","author":"Marshall","year":"2023","journal-title":"J. Manag. Care Spec. Pharm."},{"key":"10.1016\/j.ejphar.2024.176390_bib44","doi-asserted-by":"crossref","first-page":"1809","DOI":"10.1056\/NEJMoa1908639","article-title":"Elexacaftor\u2013Tezacaftor\u2013Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele","volume":"381","author":"Middleton","year":"2019","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ejphar.2024.176390_bib45","doi-asserted-by":"crossref","first-page":"444","DOI":"10.1038\/nchembio.1253","article-title":"Mechanism-based corrector combination restores \u0394F508-CFTR folding and function","volume":"9","author":"Okiyoneda","year":"2013","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.ejphar.2024.176390_bib46","first-page":"1","article-title":"The revolution of personalized pharmacotherapies for cystic fibrosis: what does the future hold?","author":"Oliver","year":"2023","journal-title":"Expet Opin. Pharmacother."},{"key":"10.1016\/j.ejphar.2024.176390_bib47","doi-asserted-by":"crossref","first-page":"5236","DOI":"10.1172\/JCI124282","article-title":"Slowing ribosome velocity restores folding and function of mutant CFTR","volume":"129","author":"Oliver","year":"2019","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ejphar.2024.176390_bib48","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1007\/s00018-018-2989-3","article-title":"Increases in cytosolic Ca 2+ induce dynamin- and calcineurin-dependent internalisation of CFTR","volume":"76","author":"Patel","year":"2019","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.ejphar.2024.176390_bib49","doi-asserted-by":"crossref","first-page":"C866","DOI":"10.1152\/ajpcell.00404.2009","article-title":"Influence of cell background on pharmacological rescue of mutant CFTR","volume":"298","author":"Pedemonte","year":"2010","journal-title":"Am. J. Physiol. Cell Physiol."},{"key":"10.1016\/j.ejphar.2024.176390_bib50","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41598-019-54158-2","article-title":"Nanomolar-potency \u2018co-potentiator\u2019 therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants","volume":"9","author":"Phuan","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ejphar.2024.176390_bib51","doi-asserted-by":"crossref","first-page":"693","DOI":"10.2147\/JEP.S255377","article-title":"Pharmacological modulation of ion channels for the treatment of cystic fibrosis","volume":"13","author":"Pinto","year":"2021","journal-title":"J. Exp. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib52","doi-asserted-by":"crossref","DOI":"10.1016\/j.jbc.2022.101615","article-title":"Can two wrongs make a right? F508del-CFTR ion channel rescue by second-site mutations in its transmembrane domains","volume":"298","author":"Prins","year":"2022","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ejphar.2024.176390_bib53","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1016\/j.cell.2011.11.024","article-title":"Correction of both NBD1 energetics and domain interface is required to restore \u0394F508 CFTR folding and function","volume":"148","author":"Rabeh","year":"2012","journal-title":"Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib54","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0119796","article-title":"Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation","volume":"10","author":"Rapino","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.ejphar.2024.176390_bib55","doi-asserted-by":"crossref","first-page":"3016","DOI":"10.1091\/mbc.e13-05-0240","article-title":"VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1","volume":"24","author":"Ren","year":"2013","journal-title":"Mol. Biol. Cell"},{"issue":"80-","key":"10.1016\/j.ejphar.2024.176390_bib56","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"key":"10.1016\/j.ejphar.2024.176390_bib57","doi-asserted-by":"crossref","first-page":"17891","DOI":"10.1073\/pnas.0608312103","article-title":"Revertant mutants G550E and 4RK rescue cystic fibrosis mutants in the first nucleotide-binding domain of CFTR by different mechanisms","volume":"103","author":"Roxo-Rosa","year":"2006","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ejphar.2024.176390_bib58","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1091\/mbc.e09-05-0427","article-title":"Interplay between ER exit code and domain conformation in CFTR misprocessing and rescue","volume":"21","author":"Roy","year":"2010","journal-title":"Mol. Biol. Cell"},{"key":"10.1016\/j.ejphar.2024.176390_bib59","doi-asserted-by":"crossref","first-page":"1321","DOI":"10.3390\/jpm12081321","article-title":"Advances in preclinical in vitro models for the translation of precision medicine for cystic fibrosis","volume":"12","author":"Silva","year":"2022","journal-title":"J. Pers. Med."},{"key":"10.1016\/j.ejphar.2024.176390_bib60","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1124\/jpet.119.261800","article-title":"Biological characterization of F508DelCFTR protein processing by the CFTR corrector ABBV-2222\/GLPG2222","volume":"372","author":"Singh","year":"2020","journal-title":"J. Pharmacol. Exp. Therapeut."},{"key":"10.1016\/j.ejphar.2024.176390_bib61","doi-asserted-by":"crossref","first-page":"C872","DOI":"10.1152\/ajpcell.00507.2010","article-title":"Rescue of the mutant CFTR chloride channel by pharmacological correctors and low temperature analyzed by gene expression profiling","volume":"301","author":"Sondo","year":"2011","journal-title":"Am. J. Physiol. Cell Physiol."},{"key":"10.1016\/j.ejphar.2024.176390_bib62","doi-asserted-by":"crossref","first-page":"35825","DOI":"10.1074\/jbc.M110.131623","article-title":"The cystic fibrosis-causing mutation deltaF508 affects multiple steps in cystic fibrosis transmembrane conductance regulator biogenesis","volume":"285","author":"Thibodeau","year":"2010","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ejphar.2024.176390_bib63","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1016\/j.jcf.2022.12.005","article-title":"Rescue by elexacaftor-tezacaftor-ivacaftor of the G1244E cystic fibrosis mutation's stability and gating defects are dependent on cell background","volume":"22","author":"Tomati","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib64","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/j.jcf.2018.12.001","article-title":"Isogenic cell models of cystic fibrosis-causing variants in natively expressing pulmonary epithelial cells","volume":"18","author":"Valley","year":"2019","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2024.176390_bib65","doi-asserted-by":"crossref","first-page":"555","DOI":"10.1042\/BJ20071420","article-title":"Enhanced cell-surface stability of rescued DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) by pharmacological chaperones","volume":"410","author":"Varga","year":"2008","journal-title":"Biochem. J."},{"key":"10.1016\/j.ejphar.2024.176390_bib66","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.139983","article-title":"Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination","volume":"5","author":"Veit","year":"2020","journal-title":"JCI Insight"},{"key":"10.1016\/j.ejphar.2024.176390_bib67","doi-asserted-by":"crossref","first-page":"1878","DOI":"10.1111\/j.1600-0854.2008.00806.x","article-title":"Chemical and biological folding contribute to temperature sensitive \u0394F508 CFTR Trafficking.pdf","volume":"9","author":"Wang","year":"2008","journal-title":"Traffic"},{"key":"10.1016\/j.ejphar.2024.176390_bib68","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1083\/jcb.200401035","article-title":"COPII-dependent export of cystic fibrosis transmembrane conductance regulator from the ER uses a di-acidic exit code","volume":"167","author":"Wang","year":"2004","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.ejphar.2024.176390_bib69","doi-asserted-by":"crossref","DOI":"10.3389\/fphar.2023.1163391","article-title":"Real-world disparities and ethical considerations with access to CFTR modulator drugs: mind the gap","volume":"14","author":"Zampoli","year":"2023","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.ejphar.2024.176390_bib70","doi-asserted-by":"crossref","first-page":"176","DOI":"10.3389\/fphar.2012.00176","article-title":"Ouabain mimics low temperature rescue of F508del-CFTR in cystic fibrosis epithelial cells","volume":"3","author":"Zhang","year":"2012","journal-title":"Front. Pharmacol."}],"container-title":["European Journal of Pharmacology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014299924000785?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014299924000785?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,15]],"date-time":"2025-09-15T23:31:38Z","timestamp":1757979098000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014299924000785"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3]]},"references-count":72,"alternative-id":["S0014299924000785"],"URL":"https:\/\/doi.org\/10.1016\/j.ejphar.2024.176390","relation":{},"ISSN":["0014-2999"],"issn-type":[{"value":"0014-2999","type":"print"}],"subject":[],"published":{"date-parts":[[2024,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR","name":"articletitle","label":"Article Title"},{"value":"European Journal of Pharmacology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ejphar.2024.176390","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 The Authors. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}],"article-number":"176390"}}