{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T05:26:53Z","timestamp":1793424413676,"version":"4.5.0"},"reference-count":74,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T00:00:00Z","timestamp":1770163200000},"content-version":"vor","delay-in-days":3,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100024370","name":"Ministero dell&apos;Istruzione dell&apos;Universita e della Ricerca","doi-asserted-by":"publisher","award":["2022FRSS2H"],"award-info":[{"award-number":["2022FRSS2H"]}],"id":[{"id":"10.13039\/501100024370","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006501","name":"Cystic Fibrosis Foundation Therapeutics Lab","doi-asserted-by":"publisher","award":["Assael08A0"],"award-info":[{"award-number":["Assael08A0"]}],"id":[{"id":"10.13039\/100006501","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":[[2026,2]]},"DOI":"10.1016\/j.ejphar.2026.178612","type":"journal-article","created":{"date-parts":[[2026,1,31]],"date-time":"2026-01-31T16:01:52Z","timestamp":1769875312000},"page":"178612","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"special_numbering":"C","title":["Comparison of preclinical drug efficacy in nasal and intestinal patient-derived models of cystic fibrosis"],"prefix":"10.1016","volume":"1016","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6722-5683","authenticated-orcid":false,"given":"Roberta Valeria","family":"Latorre","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Mortali","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Virginia","family":"Lotti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karina","family":"Kleinfelder","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paola","family":"Melotti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2739-4014","authenticated-orcid":false,"given":"Claudio","family":"Sorio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ejphar.2026.178612_bib1","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1016\/j.jcf.2022.01.008","article-title":"Forskolin induced swelling (FIS) assay in intestinal organoids to guide eligibility for compassionate use treatment in a CF patient with a rare genotype","volume":"21","author":"Aalbers","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib2","doi-asserted-by":"crossref","DOI":"10.3389\/fmolb.2023.1148501","article-title":"Q1291H-CFTR molecular dynamics simulations and ex vivo theratyping in nasal epithelial models and clinical response to elexacaftor\/tezacaftor\/ivacaftor in a Q1291H\/F508del patient","volume":"10","author":"Allan","year":"2023","journal-title":"Front. Mol. Biosci."},{"key":"10.1016\/j.ejphar.2026.178612_bib3","doi-asserted-by":"crossref","first-page":"685","DOI":"10.1016\/j.jcf.2019.06.010","article-title":"Theranostics by testing CFTR modulators in patient-derived materials: the current status and a proposal for subjects with rare CFTR mutations","volume":"18","author":"Amaral","year":"2019","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib4","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.jcf.2024.09.013","article-title":"Theranostics vs theratyping or theranostics plus theratyping?","volume":"24","author":"Amaral","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib5","doi-asserted-by":"crossref","first-page":"L119","DOI":"10.1152\/ajplung.00639.2020","article-title":"CFTR function and clinical response to modulators parallel nasal epithelial organoid swelling","volume":"321","author":"Anderson","year":"2021","journal-title":"Am. J. Physiol. Lung Cell. Mol. Physiol."},{"key":"10.1016\/j.ejphar.2026.178612_bib6","doi-asserted-by":"crossref","first-page":"1429","DOI":"10.3389\/fphar.2018.01429","article-title":"Human primary epithelial cell models: promising tools in the era of cystic fibrosis personalized medicine","volume":"9","author":"Awatade","year":"2018","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.ejphar.2026.178612_bib7","doi-asserted-by":"crossref","first-page":"156","DOI":"10.1186\/1471-2466-14-156","article-title":"Intestinal current measurement versus nasal potential difference measurements for diagnosis of cystic fibrosis: a case\u2013control study","volume":"14","author":"Bagheri-Hanson","year":"2014","journal-title":"BMC Pulm. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib8","doi-asserted-by":"crossref","DOI":"10.1002\/anbr.202000111","article-title":"Air-liquid interface cultures of the healthy and diseased human respiratory tract: promises, challenges and future directions","volume":"1","author":"Baldassi","year":"2021","journal-title":"Adv Nanobiomed Res"},{"key":"10.1016\/j.ejphar.2026.178612_bib9","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1016\/0092-8674(92)90155-6","article-title":"Purification and functional reconstitution of the cystic fibrosis transmembrane conductance regulator (CFTR)","volume":"68","author":"Bear","year":"1992","journal-title":"Cell"},{"key":"10.1016\/j.ejphar.2026.178612_bib10","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1016\/j.celrep.2019.01.068","article-title":"Rectal organoids enable personalized treatment of cystic fibrosis","volume":"26","author":"Berkers","year":"2019","journal-title":"Cell Rep."},{"key":"10.1016\/j.ejphar.2026.178612_bib11","doi-asserted-by":"crossref","DOI":"10.26508\/lsa.202201857","article-title":"Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis","volume":"6","author":"Birimberg-Schwartz","year":"2023","journal-title":"Life Sci. Alliance"},{"key":"10.1016\/j.ejphar.2026.178612_bib12","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1093\/jmcb\/mjaa034","article-title":"Intestinal organoids as tools for enriching and studying specific and rare cell types: advances and future directions","volume":"12","author":"Boonekamp","year":"2020","journal-title":"J. Mol. Cell Biol."},{"key":"10.1016\/j.ejphar.2026.178612_bib13","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1016\/j.jcf.2017.06.010","article-title":"Detection of CFTR function and modulation in primary human nasal cell spheroids","volume":"17","author":"Brewington","year":"2018","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib14","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1139\/o91-016","article-title":"Cultured human nasal epithelial multicellular spheroids: polar cyst-like model tissues","volume":"69","author":"Bridges","year":"1991","journal-title":"Biochem. Cell. Biol."},{"key":"10.1016\/j.ejphar.2026.178612_bib15","article-title":"Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells","volume":"5","author":"Bulcaen","year":"2024","journal-title":"Cell Rep. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib16","doi-asserted-by":"crossref","DOI":"10.1016\/j.ejmech.2023.116120","article-title":"Small molecule modulators of cystic fibrosis transmembrane conductance regulator (CFTR): structure, classification, and mechanisms","volume":"265","author":"Cao","year":"2024","journal-title":"Eur. J. Med. Chem."},{"key":"10.1016\/j.ejphar.2026.178612_bib17","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/j.jcf.2005.12.001","article-title":"Phenotypic discordance in three siblings affected by atypical cystic fibrosis with the F508del\/D614G genotype","volume":"5","author":"Castaldo","year":"2006","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib18","doi-asserted-by":"crossref","first-page":"632","DOI":"10.3390\/jpm12040632","article-title":"Theratyping of the rare CFTR variants E193K and R334W in rectal organoid-derived epithelial monolayers","volume":"12","author":"Ciciriello","year":"2022","journal-title":"J. Personalized Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib19","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0073905","article-title":"Multicenter intestinal current measurements in rectal biopsies from CF and non-CF subjects to monitor CFTR function","volume":"8","author":"Clancy","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.ejphar.2026.178612_bib20","doi-asserted-by":"crossref","first-page":"774","DOI":"10.1126\/science.256.5058.774","article-title":"Cystic fibrosis: molecular biology and therapeutic implications","volume":"256","author":"Collins","year":"1992","journal-title":"Science"},{"key":"10.1016\/j.ejphar.2026.178612_bib21","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1016\/j.jcf.2025.02.008","article-title":"Evaluation of the response to elexacaftor-tezacaftor-ivacaftor of the rare CFTR variants L383S, I507del, L1065P and R1066H in intestinal organoid-derived epithelial monolayers","volume":"24","author":"Conti","year":"2025","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib22","doi-asserted-by":"crossref","DOI":"10.3390\/children10010004","article-title":"Organoid technology and its role for theratyping applications in cystic fibrosis","volume":"10","author":"Conti","year":"2022","journal-title":"Children"},{"key":"10.1016\/j.ejphar.2026.178612_bib23","doi-asserted-by":"crossref","first-page":"772","DOI":"10.1513\/pats.200805-041HR","article-title":"Airway epithelial cells: current concepts and challenges","volume":"5","author":"Crystal","year":"2008","journal-title":"Proc. Am. Thorac. Soc."},{"key":"10.1016\/j.ejphar.2026.178612_bib24","doi-asserted-by":"crossref","first-page":"1146","DOI":"10.1136\/thoraxjnl-2020-216368","article-title":"Rectal organoid morphology analysis (ROMA) as a promising diagnostic tool in cystic fibrosis","volume":"76","author":"Cuyx","year":"2021","journal-title":"Thorax"},{"issue":"Suppl. 1","key":"10.1016\/j.ejphar.2026.178612_bib25","doi-asserted-by":"crossref","first-page":"S60","DOI":"10.1016\/j.jcf.2019.11.002","article-title":"Intestinal organoids for cystic fibrosis research","volume":"19","author":"de Poel","year":"2020","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib26","doi-asserted-by":"crossref","first-page":"344ra384","DOI":"10.1126\/scitranslmed.aad8278","article-title":"Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis","volume":"8","author":"Dekkers","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib27","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1038\/nm.3201","article-title":"A functional CFTR assay using primary cystic fibrosis intestinal organoids","volume":"19","author":"Dekkers","year":"2013","journal-title":"Nat. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib28","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1136\/thx.2009.125088","article-title":"Intestinal current measurement for diagnostic classification of patients with questionable cystic fibrosis: validation and reference data","volume":"65","author":"Derichs","year":"2010","journal-title":"Thorax"},{"key":"10.1016\/j.ejphar.2026.178612_bib29","doi-asserted-by":"crossref","DOI":"10.1183\/13993003.00110-2023","article-title":"Theratyping cystic fibrosis patients to guide elexacaftor\/tezacaftor\/ivacaftor out-of-label prescription","volume":"62","author":"Dreano","year":"2023","journal-title":"Eur. Respir. J."},{"key":"10.1016\/j.ejphar.2026.178612_bib30","doi-asserted-by":"crossref","DOI":"10.3390\/jpm11050384","article-title":"Potential of intestinal current measurement for personalized treatment of patients with cystic fibrosis","volume":"11","author":"Graeber","year":"2021","journal-title":"J. Personalized Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib31","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.95734","article-title":"Nasospheroids permit measurements of CFTR-dependent fluid transport","volume":"2","author":"Guimbellot","year":"2017","journal-title":"JCI Insight"},{"key":"10.1016\/j.ejphar.2026.178612_bib32","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.2026.178612_bib33","article-title":"Production of 3-D airway organoids from primary human airway basal cells and their use in high-throughput screening","volume":"37","author":"Hild","year":"2016","journal-title":"Curr Protoc Stem Cell Biol"},{"key":"10.1016\/j.ejphar.2026.178612_bib34","doi-asserted-by":"crossref","DOI":"10.3390\/ijms22094448","article-title":"Nasal epithelial cell-based models for individualized study in cystic fibrosis","volume":"22","author":"Keegan","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ejphar.2026.178612_bib35","doi-asserted-by":"crossref","DOI":"10.1016\/j.isci.2023.108180","article-title":"In silico analysis and theratyping of an ultra-rare CFTR genotype (W57G\/A234D) in primary human rectal and nasal epithelial cells","volume":"26","author":"Kleinfelder","year":"2023","journal-title":"iScience"},{"key":"10.1016\/j.ejphar.2026.178612_bib36","doi-asserted-by":"crossref","first-page":"343","DOI":"10.1186\/s13023-024-03334-3","article-title":"CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids","volume":"19","author":"Kleinfelder","year":"2024","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ejphar.2026.178612_bib37","doi-asserted-by":"crossref","first-page":"10358","DOI":"10.3390\/ijms241210358","article-title":"Theratyping of the rare CFTR genotype A559T in rectal organoids and nasal cells reveals a relevant response to elexacaftor (VX-445) and Tezacaftor (VX-661) combination","volume":"24","author":"Kleinfelder","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ejphar.2026.178612_bib38","doi-asserted-by":"crossref","first-page":"1323","DOI":"10.1016\/j.bbadis.2019.01.029","article-title":"Extent of rescue of F508del-CFTR function by VX-809 and VX-770 in human nasal epithelial cells correlates with SNP rs7512462 in SLC26A9 gene in F508del\/F508del cystic fibrosis patients","volume":"1865","author":"Kmit","year":"2019","journal-title":"Biochim. Biophys. Acta Mol. Basis Dis."},{"key":"10.1016\/j.ejphar.2026.178612_bib39","doi-asserted-by":"crossref","first-page":"229","DOI":"10.3390\/jor1040022","article-title":"Three-dimensional airway spheroids and organoids for cystic fibrosis research","volume":"1","author":"Laselva","year":"2021","journal-title":"Journal of Respiration"},{"key":"10.1016\/j.ejphar.2026.178612_bib40","doi-asserted-by":"crossref","DOI":"10.1016\/j.addr.2023.114866","article-title":"Air-liquid interface cultures to model drug delivery through the mucociliary epithelial barrier","volume":"198","author":"Lee","year":"2023","journal-title":"Adv. Drug Deliv. Rev."},{"issue":"Suppl. 2","key":"10.1016\/j.ejphar.2026.178612_bib41","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.2026.178612_bib42","doi-asserted-by":"crossref","DOI":"10.3390\/genes11060603","article-title":"Human nasal epithelial organoids for therapeutic development in cystic fibrosis","volume":"11","author":"Liu","year":"2020","journal-title":"Genes"},{"key":"10.1016\/j.ejphar.2026.178612_bib43","doi-asserted-by":"crossref","DOI":"10.3390\/cells14231914","article-title":"CFTR modulator response in nasal organoids derived from people with cystic fibrosis","volume":"14","author":"Lo Cicero","year":"2025","journal-title":"Cells"},{"key":"10.1016\/j.ejphar.2026.178612_bib44","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1165\/rcmb.2007-0325OC","article-title":"Nasal epithelial cells as surrogates for bronchial epithelial cells in airway inflammation studies","volume":"39","author":"McDougall","year":"2008","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"10.1016\/j.ejphar.2026.178612_bib45","doi-asserted-by":"crossref","first-page":"2201341","DOI":"10.1183\/13993003.01341-2022","article-title":"Rectal organoid-guided CFTR modulator therapy restores lung function in a cystic fibrosis patient with the rare 1677delTA\/R334W genotype","volume":"60","author":"Mitropoulou","year":"2022","journal-title":"Eur. Respir. J."},{"key":"10.1016\/j.ejphar.2026.178612_bib46","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1007\/978-1-0716-0747-3_11","article-title":"Primary intestinal epithelial organoid culture","volume":"2171","author":"Mizutani","year":"2020","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.ejphar.2026.178612_bib47","doi-asserted-by":"crossref","DOI":"10.3791\/50646-v","article-title":"Culturing of human nasal epithelial cells at the air liquid interface","author":"M\u00fcller","year":"2013","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.ejphar.2026.178612_bib48","doi-asserted-by":"crossref","DOI":"10.1016\/j.isci.2025.111979","article-title":"Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids","volume":"28","author":"Nicosia","year":"2025","journal-title":"iScience"},{"key":"10.1016\/j.ejphar.2026.178612_bib49","doi-asserted-by":"crossref","first-page":"610","DOI":"10.1097\/MCP.0000000000000315","article-title":"Intestinal organoids and personalized medicine in cystic fibrosis: a successful patient-oriented research collaboration","volume":"22","author":"Noordhoek","year":"2016","journal-title":"Curr. Opin. Pulm. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib50","doi-asserted-by":"crossref","first-page":"1891","DOI":"10.1016\/S0140-6736(09)60327-5","article-title":"Cystic fibrosis","volume":"373","author":"O'Sullivan","year":"2009","journal-title":"Lancet"},{"key":"10.1016\/j.ejphar.2026.178612_bib51","doi-asserted-by":"crossref","first-page":"1285","DOI":"10.1113\/JP282586","article-title":"Nasal epithelial cells as a gold-standard predictive model for personalized medicine in cystic fibrosis","volume":"600","author":"Pedemonte","year":"2022","journal-title":"J. Physiol."},{"key":"10.1016\/j.ejphar.2026.178612_bib52","doi-asserted-by":"crossref","first-page":"7375","DOI":"10.1038\/s41598-017-07504-1","article-title":"Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators","volume":"7","author":"Pranke","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ejphar.2026.178612_bib53","doi-asserted-by":"crossref","DOI":"10.1128\/JB.00311-21","article-title":"The gut-lung axis in cystic fibrosis","volume":"203","author":"Price","year":"2021","journal-title":"J. Bacteriol."},{"issue":"Suppl. 1","key":"10.1016\/j.ejphar.2026.178612_bib54","doi-asserted-by":"crossref","first-page":"S32","DOI":"10.1016\/j.jcf.2022.11.007","article-title":"Patient-derived cell models for personalized medicine approaches in cystic fibrosis","volume":"22","author":"Ramalho","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib55","doi-asserted-by":"crossref","DOI":"10.1183\/13993003.02426-2019","article-title":"Correction of CFTR function in intestinal organoids to guide treatment of cystic fibrosis","volume":"57","author":"Ramalho","year":"2021","journal-title":"Eur. Respir. J."},{"key":"10.1016\/j.ejphar.2026.178612_bib56","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-023-45201-4","article-title":"Exploring intrinsic variability between cultured nasal and bronchial epithelia in cystic fibrosis","volume":"13","author":"Rodenburg","year":"2023","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ejphar.2026.178612_bib57","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1007\/s00018-025-05587-y","article-title":"Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells","volume":"82","author":"Rose","year":"2025","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.ejphar.2026.178612_bib58","series-title":"Cystic Fibrosis: Diagnosis and Protocols, Volume I: Approaches to Study and Correct CFTR Defects","first-page":"69","article-title":"Nasal potential difference measurements to assess CFTR ion channel activity","author":"Rowe","year":"2011"},{"key":"10.1016\/j.ejphar.2026.178612_bib59","doi-asserted-by":"crossref","first-page":"1062","DOI":"10.1016\/j.jcf.2023.06.001","article-title":"Clinical and functional efficacy of elexacaftor\/tezacaftor\/ivacaftor in people with cystic fibrosis carrying the N1303K mutation","volume":"22","author":"Sadras","year":"2023","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib60","doi-asserted-by":"crossref","first-page":"1190","DOI":"10.1126\/science.1234852","article-title":"Growing self-organizing mini-guts from a single intestinal stem cell: mechanism and applications","volume":"340","author":"Sato","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.ejphar.2026.178612_bib61","doi-asserted-by":"crossref","first-page":"262","DOI":"10.1038\/nature07935","article-title":"Single Lgr5 stem cells build crypt-villus structures in vitro without a mesenchymal niche","volume":"459","author":"Sato","year":"2009","journal-title":"Nature"},{"key":"10.1016\/j.ejphar.2026.178612_bib62","first-page":"127","article-title":"Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives","volume":"8","author":"Schmidt","year":"2016","journal-title":"Clin. Pharmacol."},{"key":"10.1016\/j.ejphar.2026.178612_bib63","doi-asserted-by":"crossref","DOI":"10.3390\/cells9092090","article-title":"Ionocytes and CFTR chloride channel expression in normal and cystic fibrosis nasal and bronchial epithelial cells","volume":"9","author":"Scudieri","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.ejphar.2026.178612_bib64","doi-asserted-by":"crossref","first-page":"922","DOI":"10.1016\/j.jcf.2022.09.005","article-title":"ECFS standards of care on CFTR-related disorders: diagnostic criteria of CFTR dysfunction","volume":"21","author":"Sermet-Gaudelus","year":"2022","journal-title":"J. Cyst. Fibros."},{"key":"10.1016\/j.ejphar.2026.178612_bib65","doi-asserted-by":"crossref","DOI":"10.1183\/13993003.00908-2021","article-title":"Theratyping cystic fibrosis in vitro in ALI culture and organoid models generated from patient-derived nasal epithelial conditionally reprogrammed stem cells","volume":"58","author":"Sette","year":"2021","journal-title":"Eur. Respir. J."},{"key":"10.1016\/j.ejphar.2026.178612_bib66","doi-asserted-by":"crossref","first-page":"421","DOI":"10.3390\/jpm11050421","article-title":"Personalized medicine based on nasal epithelial cells: comparative studies with rectal biopsies and intestinal organoids","volume":"11","author":"Silva","year":"2021","journal-title":"J. Personalized Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib67","doi-asserted-by":"crossref","DOI":"10.3390\/ijms24076576","article-title":"Clinical consequences and functional impact of the rare S737F CFTR variant and its responsiveness to CFTR modulators","volume":"24","author":"Terlizzi","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ejphar.2026.178612_bib68","doi-asserted-by":"crossref","first-page":"604","DOI":"10.1111\/joim.13075","article-title":"Airway organoids as models of human disease","volume":"289","author":"van der Vaart","year":"2021","journal-title":"J. Intern. Med."},{"key":"10.1016\/j.ejphar.2026.178612_bib69","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1091\/mbc.e14-04-0935","article-title":"From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations","volume":"27","author":"Veit","year":"2016","journal-title":"Mol. Biol. Cell"},{"key":"10.1016\/j.ejphar.2026.178612_bib70","doi-asserted-by":"crossref","DOI":"10.1016\/j.xpro.2020.100019","article-title":"Protocol for application, standardization and validation of the forskolin-induced swelling assay in cystic fibrosis human Colon organoids","volume":"1","author":"Vonk","year":"2020","journal-title":"STAR Protoc."},{"key":"10.1016\/j.ejphar.2026.178612_bib71","doi-asserted-by":"crossref","first-page":"336","DOI":"10.1038\/s41392-022-01194-6","article-title":"Applications of human organoids in the personalized treatment for digestive diseases","volume":"7","author":"Wang","year":"2022","journal-title":"Signal Transduct. Targeted Ther."},{"key":"10.1016\/j.ejphar.2026.178612_bib72","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1165\/rcmb.2021-0337OC","article-title":"Molecular dynamics and theratyping in airway and gut organoids reveal R352Q-CFTR conductance defect","volume":"67","author":"Wong","year":"2022","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"10.1016\/j.ejphar.2026.178612_bib73","doi-asserted-by":"crossref","DOI":"10.3390\/cells12070997","article-title":"Passaging primary human bronchial epithelia reduces CFTR-mediated fluid transport and alters mRNA expression","volume":"12","author":"Wu","year":"2023","journal-title":"Cells"},{"key":"10.1016\/j.ejphar.2026.178612_bib74","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1016\/j.jcf.2018.02.007","article-title":"Comparison of ex vivo and in vitro intestinal cystic fibrosis models to measure CFTR-Dependent ion channel activity","volume":"17","author":"Zomer-van Ommen","year":"2018","journal-title":"J. Cyst. Fibros."}],"container-title":["European Journal of Pharmacology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014299926000944?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014299926000944?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T12:05:54Z","timestamp":1774008354000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014299926000944"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,2]]},"references-count":74,"alternative-id":["S0014299926000944"],"URL":"https:\/\/doi.org\/10.1016\/j.ejphar.2026.178612","relation":{},"ISSN":["0014-2999"],"issn-type":[{"value":"0014-2999","type":"print"}],"subject":[],"published":{"date-parts":[[2026,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Comparison of preclinical drug efficacy in nasal and intestinal patient-derived models of cystic fibrosis","name":"articletitle","label":"Article Title"},{"value":"European Journal of Pharmacology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ejphar.2026.178612","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}],"article-number":"178612"}}