{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T02:07:52Z","timestamp":1793844472157,"version":"4.6.1"},"reference-count":175,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,1,1]],"date-time":"2025-01-01T00:00:00Z","timestamp":1735689600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ther Adv Respir Dis"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:p>Over the past decade, major clinical advances have been made in the healthcare and therapeutic development for cystic fibrosis (CF), a lethal genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. CFTR modulators represent innovative treatments that directly target the primary defects in the mutated CFTR protein and have demonstrated significant clinical benefits for many people with CF (pwCF) who are eligible for these treatments. In particular, the triple combination therapy composed of elexacaftor, tezacaftor, and ivacaftor (ETI) has changed the CF therapeutic landscape by significantly improving lung function, quality of life, and predicted survival rates. Here, we provided a comprehensive summary of the impact of ETI on clinical outcomes and the need for further research on long-term efficacy, side effects, pregnancy, possible drug\u2013drug interactions, and extra-pulmonary manifestations. Moreover, a significant number of pwCF are unresponsive to these drugs or cannot afford their high costs. We, therefore, discussed health inequity issues and alternative therapeutic strategies under development aiming to obtain effective therapies for all pwCF.<\/jats:p>","DOI":"10.1177\/17534666251323194","type":"journal-article","created":{"date-parts":[[2025,3,31]],"date-time":"2025-03-31T14:35:59Z","timestamp":1743431759000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":33,"title":["Cystic fibrosis: new challenges and perspectives beyond elexacaftor\/tezacaftor\/ivacaftor"],"prefix":"10.1177","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1106-4424","authenticated-orcid":false,"given":"Vito","family":"Terlizzi","sequence":"first","affiliation":[{"name":"Department of Pediatric Medicine, Cystic Fibrosis Regional Reference Center, Meyer Children\u2019s Hospital IRCCS, Viale Gaetano Pieraccini 24, Florence, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miqu\u00e9ias","family":"Lopes-Pacheco","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Cystic Fibrosis and Airway Disease Research Center, Emory University School of Medicine, Atlanta, GA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,3,31]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra2216474"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.2475911"},{"key":"e_1_3_4_4_2","unstructured":"ECFSPR 2022 Annual Data Report"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.01.009"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2019.01662"},{"key":"e_1_3_4_7_2","doi-asserted-by":"publisher","DOI":"10.1038\/nrg3849"},{"key":"e_1_3_4_8_2","doi-asserted-by":"publisher","DOI":"10.1091\/mbc.e14-04-0935"},{"key":"e_1_3_4_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(90)90148-8"},{"key":"e_1_3_4_10_2","doi-asserted-by":"publisher","DOI":"10.1038\/354526a0"},{"key":"e_1_3_4_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0021-9258(20)80582-1"},{"key":"e_1_3_4_12_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90241-4"},{"key":"e_1_3_4_13_2","article-title":"Diagnosis of cystic fibrosis: consensus guidelines from the cystic fibrosis foundation","author":"Farrell PM","year":"2017","unstructured":"Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the cystic fibrosis foundation. J Pediatr 2017; 181S: S4-S15.e1.","journal-title":"J Pediatr"},{"key":"e_1_3_4_14_2","unstructured":"Cystic Fibrosis Foundation Patient Registry Highlights 2023."},{"key":"e_1_3_4_15_2","doi-asserted-by":"publisher","DOI":"10.1055\/s-0042-1759881"},{"key":"e_1_3_4_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcte.2024.100344"},{"key":"e_1_3_4_17_2","first-page":"101637","article-title":"Update on advances in cystic fibrosis toward a cure and implications for primary care clinicians","volume":"2024","author":"Terlizzi V","unstructured":"Terlizzi V, Farrell PM. Update on advances in cystic fibrosis toward a cure and implications for primary care clinicians. Curr Probl Pediatr Adolesc Health Care 2024: 101637.","journal-title":"Curr Probl Pediatr Adolesc Health Care"},{"key":"e_1_3_4_18_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13052-022-01331-5"},{"key":"e_1_3_4_19_2","doi-asserted-by":"crossref","DOI":"10.1136\/bmjresp-2023-002049","article-title":"Considerations for the use of inhaled antibiotics for Pseudomonas aeruginosa in people with cystic fibrosis receiving CFTR modulator therapy","volume":"11","author":"Burgel PR","year":"2024","unstructured":"Burgel PR, Ballmann M, Drevinek P, et al. Considerations for the use of inhaled antibiotics for Pseudomonas aeruginosa in people with cystic fibrosis receiving CFTR modulator therapy. BMJ Open Respir Res 2024; 11: e002049.","journal-title":"BMJ Open Respir Res"},{"key":"e_1_3_4_20_2","doi-asserted-by":"publisher","DOI":"10.1080\/14656566.2023.2230129"},{"key":"e_1_3_4_21_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0904709106"},{"key":"e_1_3_4_22_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1105185"},{"key":"e_1_3_4_23_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2014.09.005"},{"key":"e_1_3_4_24_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.23659"},{"key":"e_1_3_4_25_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2018.04.004"},{"key":"e_1_3_4_26_2","unstructured":"Kalydeco (Ivacaftor) prescribing information. Boston MA: Vertex Pharmaceuticals Incorporated 2020."},{"key":"e_1_3_4_27_2","doi-asserted-by":"publisher","DOI":"10.1513\/AnnalsATS.201708-668PS"},{"key":"e_1_3_4_28_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202008-3177OC"},{"issue":"11","key":"e_1_3_4_29_2","article-title":"Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)","volume":"11","author":"Heneghan M","year":"2023","unstructured":"Heneghan M, Southern KW, Murphy J, et al. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev 2023; 11(11): CD010966.","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_3_4_30_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1409547"},{"key":"e_1_3_4_31_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201710-1983OC"},{"key":"e_1_3_4_32_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40360-022-00624-z"},{"key":"e_1_3_4_33_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1709846"},{"key":"e_1_3_4_34_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1709847"},{"key":"e_1_3_4_35_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32597-8"},{"key":"e_1_3_4_36_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1908639"},{"key":"e_1_3_4_37_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202308-1317OC"},{"key":"e_1_3_4_38_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.10.001"},{"key":"e_1_3_4_39_2","doi-asserted-by":"publisher","DOI":"10.3390\/children10020252"},{"key":"e_1_3_4_40_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(23)00324-7"},{"key":"e_1_3_4_41_2","doi-asserted-by":"publisher","DOI":"10.3390\/ph16030410"},{"key":"e_1_3_4_42_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.02.004"},{"key":"e_1_3_4_43_2","doi-asserted-by":"publisher","DOI":"10.1513\/AnnalsATS.202312-1065OC"},{"key":"e_1_3_4_44_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.chest.2022.07.004"},{"key":"e_1_3_4_45_2","doi-asserted-by":"publisher","DOI":"10.1177\/21501319231173811"},{"key":"e_1_3_4_46_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.rmed.2021.106646"},{"key":"e_1_3_4_47_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202011-4153OC"},{"key":"e_1_3_4_48_2","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9092706"},{"key":"e_1_3_4_49_2","doi-asserted-by":"publisher","DOI":"10.1007\/s10096-023-04648-z"},{"key":"e_1_3_4_50_2","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2022.914790"},{"key":"e_1_3_4_51_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.09.010"},{"key":"e_1_3_4_52_2","doi-asserted-by":"publisher","DOI":"10.14309\/ajg.0000000000001527"},{"key":"e_1_3_4_53_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.03.011"},{"key":"e_1_3_4_54_2","doi-asserted-by":"publisher","DOI":"10.1002\/rcr2.1172"},{"key":"e_1_3_4_55_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.01794-2023"},{"key":"e_1_3_4_56_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.clnesp.2023.08.028"},{"key":"e_1_3_4_57_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.05.002"},{"key":"e_1_3_4_58_2","doi-asserted-by":"publisher","DOI":"10.1128\/mbio.01935-23"},{"key":"e_1_3_4_59_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.05.002"},{"key":"e_1_3_4_60_2","doi-asserted-by":"publisher","DOI":"10.1165\/rcmb.2019-0032OC"},{"key":"e_1_3_4_61_2","doi-asserted-by":"publisher","DOI":"10.1165\/rcmb.2015-0322LE"},{"key":"e_1_3_4_62_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22083928"},{"key":"e_1_3_4_63_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.03.012"},{"key":"e_1_3_4_64_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2019.08.021"},{"key":"e_1_3_4_65_2","doi-asserted-by":"publisher","DOI":"10.1165\/rcmb.2024-0103OC"},{"key":"e_1_3_4_66_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.10.019"},{"key":"e_1_3_4_67_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ccm.2022.06.009"},{"key":"e_1_3_4_68_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.27125"},{"key":"e_1_3_4_69_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26938"},{"key":"e_1_3_4_70_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2024.114176"},{"key":"e_1_3_4_71_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202202-0392OC"},{"key":"e_1_3_4_72_2","doi-asserted-by":"publisher","DOI":"10.1136\/thoraxjnl-2020-216085"},{"key":"e_1_3_4_73_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.04.013"},{"key":"e_1_3_4_74_2","unstructured":"Kaftrio European Medicines Agency https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/kaftrio"},{"key":"e_1_3_4_75_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.23389"},{"key":"e_1_3_4_76_2","doi-asserted-by":"publisher","DOI":"10.3390\/jpm12091421"},{"key":"e_1_3_4_77_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms23063175"},{"key":"e_1_3_4_78_2","doi-asserted-by":"publisher","DOI":"10.3390\/biomedicines11020628"},{"key":"e_1_3_4_79_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.01.007"},{"key":"e_1_3_4_80_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.12.010"},{"key":"e_1_3_4_81_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-024-68511-7"},{"key":"e_1_3_4_82_2","doi-asserted-by":"publisher","DOI":"10.3389\/fmolb.2023.1327958"},{"key":"e_1_3_4_83_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202303-0380LE"},{"key":"e_1_3_4_84_2","doi-asserted-by":"publisher","DOI":"10.1097\/MCP.0000000000001014"},{"key":"e_1_3_4_85_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(24)01134-6"},{"key":"e_1_3_4_86_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26673"},{"key":"e_1_3_4_87_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(22)00504-5"},{"key":"e_1_3_4_88_2","article-title":"Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials","author":"Keating C","year":"2024","unstructured":"Keating C, Yonker LM, Vermeulen F, et al. Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials. Lancet Respir Med 2024: S2213-2600(24)00411-9.","journal-title":"Lancet Respir Med"},{"key":"e_1_3_4_89_2","article-title":"Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial","author":"Hoppe JE","year":"2024","unstructured":"Hoppe JE, Kasi AS, Pittman JE, et al. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med 2024: S2213-2600(24)00407-7.","journal-title":"Lancet Respir Med"},{"key":"e_1_3_4_90_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.10.002"},{"key":"e_1_3_4_91_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.02029-2022"},{"key":"e_1_3_4_92_2","doi-asserted-by":"publisher","DOI":"10.1136\/thoraxjnl-2020-215556"},{"key":"e_1_3_4_93_2","doi-asserted-by":"publisher","DOI":"10.1136\/thoraxjnl-2021-216974"},{"key":"e_1_3_4_94_2","first-page":"26","article-title":"The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial","volume":"30","author":"Gold LS","year":"2024","unstructured":"Gold LS, Hansen RN, Mayer-Hamblett N, et al. The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial. J Manag Care Spec Pharm 2024; 30: 26\u201333.","journal-title":"J Manag Care Spec Pharm"},{"key":"e_1_3_4_95_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(22)00434-9"},{"key":"e_1_3_4_96_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.02.003"},{"key":"e_1_3_4_97_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201609-1954OC"},{"key":"e_1_3_4_98_2","doi-asserted-by":"publisher","DOI":"10.1128\/mbio.00519-24"},{"key":"e_1_3_4_99_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI167957"},{"key":"e_1_3_4_100_2","doi-asserted-by":"publisher","DOI":"10.1183\/16000617.0154-2022"},{"key":"e_1_3_4_101_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.10.004"},{"key":"e_1_3_4_102_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijns9010010"},{"key":"e_1_3_4_103_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.03.018"},{"key":"e_1_3_4_104_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.04.005"},{"key":"e_1_3_4_105_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.27060"},{"key":"e_1_3_4_106_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2023.1163391"},{"key":"e_1_3_4_107_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.09.003"},{"key":"e_1_3_4_108_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-024-00538-6"},{"key":"e_1_3_4_109_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26954"},{"key":"e_1_3_4_110_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2020.07.017"},{"key":"e_1_3_4_111_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-022-02350-5"},{"key":"e_1_3_4_112_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.08.002"},{"key":"e_1_3_4_113_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.01146-2024"},{"key":"e_1_3_4_114_2","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(24)00208-X"},{"key":"e_1_3_4_115_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.02437-2022"},{"key":"e_1_3_4_116_2","unstructured":"Registro Brasileiro De Fibrose C\u00edstica \u2013 REBRAFC http:\/\/portalgbefc.org.br\/ckfinder\/userfiles\/files\/Rebrafc_2021_REV_fev24.pdf"},{"key":"e_1_3_4_117_2","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.25859"},{"key":"e_1_3_4_118_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.04.006"},{"key":"e_1_3_4_119_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.09.012"},{"key":"e_1_3_4_120_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.04.007"},{"key":"e_1_3_4_121_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1215982110"},{"key":"e_1_3_4_122_2","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.139983"},{"key":"e_1_3_4_123_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms22105262"},{"key":"e_1_3_4_124_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejphar.2024.176390"},{"key":"e_1_3_4_125_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201910-1943SO"},{"key":"e_1_3_4_126_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.06.002"},{"key":"e_1_3_4_127_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201503-0578OC"},{"key":"e_1_3_4_128_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2018.05.004"},{"key":"e_1_3_4_129_2","doi-asserted-by":"publisher","DOI":"10.3390\/jpm12081321"},{"key":"e_1_3_4_130_2","doi-asserted-by":"publisher","DOI":"10.3390\/jpm14010093"},{"key":"e_1_3_4_131_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202004-1200LE"},{"key":"e_1_3_4_132_2","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2020.00536"},{"key":"e_1_3_4_133_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.01341-2022"},{"key":"e_1_3_4_134_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms24076576"},{"key":"e_1_3_4_135_2","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.00110-2023"},{"key":"e_1_3_4_136_2","doi-asserted-by":"publisher","DOI":"10.3390\/genes14091705"},{"key":"e_1_3_4_137_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms241914539"},{"key":"e_1_3_4_138_2","doi-asserted-by":"publisher","DOI":"10.3390\/genes12081178"},{"key":"e_1_3_4_139_2","doi-asserted-by":"publisher","DOI":"10.3390\/antibiotics10070828"},{"key":"e_1_3_4_140_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2020.02.006"},{"key":"e_1_3_4_141_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202303-0458OC"},{"key":"e_1_3_4_142_2","doi-asserted-by":"publisher","DOI":"10.1126\/sciadv.aay9669"},{"key":"e_1_3_4_143_2","doi-asserted-by":"publisher","DOI":"10.3390\/cells11010136"},{"key":"e_1_3_4_144_2","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.4c00685"},{"key":"e_1_3_4_145_2","doi-asserted-by":"publisher","DOI":"10.1126\/sciadv.adk1814"},{"key":"e_1_3_4_146_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-004-4422-3"},{"key":"e_1_3_4_147_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2018.05.010"},{"key":"e_1_3_4_148_2","doi-asserted-by":"publisher","DOI":"10.1111\/bph.15709"},{"key":"e_1_3_4_149_2","doi-asserted-by":"publisher","DOI":"10.3390\/jpm13010102"},{"key":"e_1_3_4_150_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.01.009"},{"key":"e_1_3_4_151_2","doi-asserted-by":"publisher","DOI":"10.1152\/ajplung.00038.2023"},{"key":"e_1_3_4_152_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2024.07.017"},{"key":"e_1_3_4_153_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-021-24575-x"},{"key":"e_1_3_4_154_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-019-08329-4"},{"key":"e_1_3_4_155_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2022.04.033"},{"key":"e_1_3_4_156_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41586-023-06133-1"},{"key":"e_1_3_4_157_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1705346"},{"key":"e_1_3_4_158_2","first-page":"7454","article-title":"Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells","volume":"48","author":"Michaels WE","year":"2020","unstructured":"Michaels WE, Bridges RJ, Hastings ML. Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells. Nucleic Acids Res 2020; 48: 7454\u20137467.","journal-title":"Nucleic Acids Res"},{"key":"e_1_3_4_159_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.06.003"},{"key":"e_1_3_4_160_2","article-title":"Exon-skipping antisense oligonucleotides for cystic fibrosis therapy","volume":"119","author":"Kim YJ","year":"2022","unstructured":"Kim YJ, Sivetz N, Layne J, et al. Exon-skipping antisense oligonucleotides for cystic fibrosis therapy. Proc Natl Acad Sci USA 2022; 119: e2114858118.","journal-title":"Proc Natl Acad Sci USA"},{"key":"e_1_3_4_161_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201505-0914OC"},{"key":"e_1_3_4_162_2","doi-asserted-by":"publisher","DOI":"10.1136\/thoraxjnl-2016-208406"},{"key":"e_1_3_4_163_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41565-018-0358-x"},{"key":"e_1_3_4_164_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.04.008"},{"key":"e_1_3_4_165_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2021.02.020"},{"key":"e_1_3_4_166_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2021.03.023"},{"key":"e_1_3_4_167_2","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2024.063"},{"key":"e_1_3_4_168_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.stem.2019.11.002"},{"key":"e_1_3_4_169_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2020.04.021"},{"key":"e_1_3_4_170_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2021.05.014"},{"key":"e_1_3_4_171_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.stem.2020.01.019"},{"key":"e_1_3_4_172_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41587-020-0561-9"},{"key":"e_1_3_4_173_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkab788"},{"key":"e_1_3_4_174_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-022-28656-3"},{"key":"e_1_3_4_175_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41596-022-00724-4"},{"key":"e_1_3_4_176_2","doi-asserted-by":"publisher","DOI":"10.2147\/JEP.S255377"}],"container-title":["Therapeutic Advances in Respiratory Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/17534666251323194","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/17534666251323194","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/17534666251323194","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,6]],"date-time":"2026-01-06T08:19:29Z","timestamp":1767687569000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/17534666251323194"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1]]},"references-count":175,"alternative-id":["10.1177\/17534666251323194"],"URL":"https:\/\/doi.org\/10.1177\/17534666251323194","relation":{"has-review":[{"id-type":"doi","id":"10.1177\/17534666251323194\/v3\/response1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v2\/decision1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v1\/review2","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v1\/review1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v1\/review4","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v1\/review3","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v2\/response1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v1\/decision1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v2\/review3","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v2\/review2","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v2\/review1","asserted-by":"object"},{"id-type":"doi","id":"10.1177\/17534666251323194\/v3\/decision1","asserted-by":"object"}]},"ISSN":["1753-4666","1753-4666"],"issn-type":[{"value":"1753-4666","type":"print"},{"value":"1753-4666","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1]]},"article-number":"17534666251323194"}}