{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T20:35:21Z","timestamp":1791232521994,"version":"4.1.0"},"reference-count":55,"publisher":"Oxford University Press (OUP)","issue":"4","license":[{"start":{"date-parts":[[2014,4,1]],"date-time":"2014-04-01T00:00:00Z","timestamp":1396310400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2014,4,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>New drugs are needed to enhance premature termination codon (PTC) suppression to treat the underlying cause of cystic fibrosis (CF) and other diseases caused by nonsense mutations. We tested new synthetic aminoglycoside derivatives expressly developed for PTC suppression in a series of complementary CF models. Using a dual-luciferase reporter system containing the four most prevalent CF transmembrane conductance regulator (CFTR) nonsense mutations (G542X, R553X, R1162X, and W1282X) within their local sequence contexts (the three codons on either side of the PTC), we found that NB124 promoted the most readthrough of G542X, R1162X, and W1282X PTCs. NB124 also restored full-length CFTR expression and chloride transport in Fischer rat thyroid cells stably transduced with a CFTR\u2013G542XcDNA transgene, and was superior to gentamicin and other aminoglycosides tested. NB124 restored CFTR function to roughly 7% of wild-type activity in primary human bronchial epithelial (HBE) CF cells (G542X\/delF508), a highly relevant preclinical model with endogenous CFTR expression. Efficacy was further enhanced by addition of the CFTR potentiator, ivacaftor (VX-770), to airway cells expressing CFTR PTCs. NB124 treatment rescued CFTR function in a CF mouse model expressing a human CFTR-G542X transgene; efficacy was superior to gentamicin and exhibited favorable pharmacokinetic properties, suggesting that in vitro results translated to clinical benefit in vivo. NB124 was also less cytotoxic than gentamicin in a tissue-based model for ototoxicity. These results provide evidence that NB124 and other synthetic aminoglycosides provide a 10-fold improvement in therapeutic index over gentamicin and other first-generation aminoglycosides, providing a promising treatment for a wide array of CFTR nonsense mutations.<\/jats:p>","DOI":"10.1165\/rcmb.2013-0282oc","type":"journal-article","created":{"date-parts":[[2013,11,19]],"date-time":"2013-11-19T10:21:06Z","timestamp":1384856466000},"page":"805-816","source":"Crossref","is-referenced-by-count":153,"title":["Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor"],"prefix":"10.1093","volume":"50","author":[{"given":"Xiaojiao","family":"Xue","sequence":"first","affiliation":[{"name":"Departments of Genetics"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Venkateshwar","family":"Mutyam","sequence":"additional","affiliation":[{"name":"Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liping","family":"Tang","sequence":"additional","affiliation":[{"name":"Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silpak","family":"Biswas","sequence":"additional","affiliation":[{"name":"Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ming","family":"Du","sequence":"additional","affiliation":[{"name":"Microbiology, and"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura A.","family":"Jackson","sequence":"additional","affiliation":[{"name":"Medicine"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanying","family":"Dai","sequence":"additional","affiliation":[{"name":"Microbiology, and"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valery","family":"Belakhov","sequence":"additional","affiliation":[{"name":"the Edith and Joseph Fischer Enzyme Inhibitors Laboratory, Schulich Faculty of Chemistry, Technion\u2013Israel Institute of Technology, Haifa, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moran","family":"Shalev","sequence":"additional","affiliation":[{"name":"the Edith and Joseph Fischer Enzyme Inhibitors Laboratory, Schulich Faculty of Chemistry, Technion\u2013Israel Institute of Technology, Haifa, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fuquan","family":"Chen","sequence":"additional","affiliation":[{"name":"Kresge Hearing Research Institute, Department of Otolaryngology, University of Michigan Medical School, Ann Arbor, Michigan; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jochen","family":"Schacht","sequence":"additional","affiliation":[{"name":"Kresge Hearing Research Institute, Department of Otolaryngology, University of Michigan Medical School, Ann Arbor, Michigan; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"J. Bridges","sequence":"additional","affiliation":[{"name":"Department of Physiology and Biophysics, Rosalind Franklin University, North Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timor","family":"Baasov","sequence":"additional","affiliation":[{"name":"the Edith and Joseph Fischer Enzyme Inhibitors Laboratory, Schulich Faculty of Chemistry, Technion\u2013Israel Institute of Technology, Haifa, Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeong","family":"Hong","sequence":"additional","affiliation":[{"name":"Cell Developmental and Integrative Biology, and"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David M.","family":"Bedwell","sequence":"additional","affiliation":[{"name":"Departments of Genetics"},{"name":"Cell Developmental and Integrative Biology, and"},{"name":"Microbiology, and"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven M.","family":"Rowe","sequence":"additional","affiliation":[{"name":"Medicine"},{"name":"Pediatrics"},{"name":"Cell Developmental and Integrative Biology, and"},{"name":"Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2014,4,1]]},"reference":[{"key":"2026030623190975300_bib1","doi-asserted-by":"crossref","first-page":"1992","DOI":"10.1056\/NEJMra043184","article-title":"Cystic fibrosis","volume":"352","author":"Rowe","year":"2005","journal-title":"N Engl J Med"},{"key":"2026030623190975300_bib2","doi-asserted-by":"crossref","first-page":"1517","DOI":"10.1056\/NEJM199011293232203","article-title":"The relation between genotype and phenotype in cystic fibrosis\u2014analysis of the most common mutation (delta F508)","volume":"323","author":"Kerem","year":"1990","journal-title":"N Engl J Med"},{"key":"2026030623190975300_bib3","doi-asserted-by":"crossref","first-page":"444","DOI":"10.3109\/10409238.2012.694846","article-title":"Suppression of premature termination codons as a therapeutic approach","volume":"47","author":"Keeling","year":"2012","journal-title":"Crit Rev Biochem Mol Biol"},{"key":"2026030623190975300_bib4","doi-asserted-by":"crossref","first-page":"1280","DOI":"10.1038\/nm1197-1280","article-title":"Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line","volume":"3","author":"Bedwell","year":"1997","journal-title":"Nat Med"},{"key":"2026030623190975300_bib5","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1038\/nm0496-467","article-title":"Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations","volume":"2","author":"Howard","year":"1996","journal-title":"Nat Med"},{"key":"2026030623190975300_bib6","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1007\/s00109-002-0363-1","article-title":"Aminoglycoside suppression of a premature stop mutation in a Cftr\u2212\/\u2212 mouse carrying a human CFTR-G542X transgene","volume":"80","author":"Du","year":"2002","journal-title":"J Mol Med (Berl)"},{"key":"2026030623190975300_bib7","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1172\/JCI7866","article-title":"Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of MDX mice","volume":"104","author":"Barton-Davis","year":"1999","journal-title":"J Clin Invest"},{"key":"2026030623190975300_bib8","doi-asserted-by":"crossref","first-page":"1683","DOI":"10.1164\/ajrccm.163.7.2004001","article-title":"Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis","volume":"163","author":"Clancy","year":"2001","journal-title":"Am J Respir Crit Care Med"},{"key":"2026030623190975300_bib9","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1164\/ajrccm.161.3.9904116","article-title":"A pilot study of the effect of gentamicin on nasal potential difference measurements in cystic fibrosis patients carrying stop mutations","volume":"161","author":"Wilschanski","year":"2000","journal-title":"Am J Respir Crit Care Med"},{"key":"2026030623190975300_bib10","doi-asserted-by":"crossref","first-page":"1433","DOI":"10.1056\/NEJMoa022170","article-title":"Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations","volume":"349","author":"Wilschanski","year":"2003","journal-title":"N Engl J Med"},{"key":"2026030623190975300_bib11","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1186\/1741-7015-5-5","article-title":"In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study","volume":"5","author":"Sermet-Gaudelus","year":"2007","journal-title":"BMC Med"},{"key":"2026030623190975300_bib12","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1159\/000013861","article-title":"Aminoglycoside antibiotics","volume":"5","author":"Forge","year":"2000","journal-title":"Audiol Neurootol"},{"key":"2026030623190975300_bib13","doi-asserted-by":"crossref","first-page":"6885","DOI":"10.1074\/jbc.M806728200","article-title":"Poly-L-aspartic acid enhances and prolongs gentamicin-mediated suppression of the CFTR-G542X mutation in a cystic fibrosis mouse model","volume":"284","author":"Du","year":"2009","journal-title":"J Biol Chem"},{"key":"2026030623190975300_bib14","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/nature05756","article-title":"PTC124 targets genetic disorders caused by nonsense mutations","volume":"447","author":"Welch","year":"2007","journal-title":"Nature"},{"key":"2026030623190975300_bib15","doi-asserted-by":"crossref","first-page":"2064","DOI":"10.1073\/pnas.0711795105","article-title":"PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model","volume":"105","author":"Du","year":"2008","journal-title":"Proc Natl Acad Sci USA"},{"key":"2026030623190975300_bib16","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1183\/09031936.00120910","article-title":"Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis","volume":"38","author":"Wilschanski","year":"2011","journal-title":"Eur Respir J"},{"key":"2026030623190975300_bib17","doi-asserted-by":"crossref","first-page":"719","DOI":"10.1016\/S0140-6736(08)61168-X","article-title":"Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial","volume":"372","author":"Kerem","year":"2008","journal-title":"Lancet"},{"key":"2026030623190975300_bib18","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1164\/rccm.201001-0137OC","article-title":"Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis","volume":"182","author":"Sermet-Gaudelus","year":"2010","journal-title":"Am J Respir Crit Care Med"},{"key":"2026030623190975300_bib19","article-title":"Results of the phase 3 study of ataluren in nonsense mutation cystic fibrosis (NMCF)","volume":"11","author":"Konstan","year":"2012","journal-title":"J Cyst Fibros"},{"key":"2026030623190975300_bib20","doi-asserted-by":"crossref","first-page":"2836","DOI":"10.1021\/jm801640k","article-title":"Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations","volume":"52","author":"Nudelman","year":"2009","journal-title":"J Med Chem"},{"key":"2026030623190975300_bib21","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1007\/s00109-011-0787-6","article-title":"Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54","volume":"89","author":"Rowe","year":"2011","journal-title":"J Mol Med (Berl)"},{"key":"2026030623190975300_bib22","doi-asserted-by":"crossref","first-page":"10630","DOI":"10.1021\/jm3012992","article-title":"Increased selectivity toward cytoplasmic versus mitochondrial ribosome confers improved efficiency of synthetic aminoglycosides in fixing damaged genes: a strategy for treatment of genetic diseases caused by nonsense mutations","volume":"55","author":"Kandasamy","year":"2012","journal-title":"J Med Chem"},{"key":"2026030623190975300_bib23","first-page":"479","article-title":"A dual-luciferase reporter system for studying recoding signals","volume":"4","author":"Grentzmann","year":"1998","journal-title":"RNA"},{"key":"2026030623190975300_bib24","doi-asserted-by":"crossref","first-page":"164","DOI":"10.1002\/1531-8249(200008)48:2<164::AID-ANA5>3.0.CO;2-B","article-title":"Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy","volume":"48","author":"Howard","year":"2000","journal-title":"Ann Neurol"},{"key":"2026030623190975300_bib25","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1016\/j.jcf.2011.12.005","article-title":"Ivacaftor potentiation of multiple CFTR channels with gating mutations","volume":"11","author":"Yu","year":"2012","journal-title":"J Cyst Fibros"},{"key":"2026030623190975300_bib26","doi-asserted-by":"crossref","first-page":"e39809","DOI":"10.1371\/journal.pone.0039809","article-title":"A pharmacologic approach to acquired cystic fibrosis transmembrane conductance regulator dysfunction in smoking related lung disease","volume":"7","author":"Sloane","year":"2012","journal-title":"PLoS ONE"},{"key":"2026030623190975300_bib27","doi-asserted-by":"crossref","first-page":"L587","DOI":"10.1152\/ajplung.00465.2010","article-title":"Regulatory domain phosphorylation to distinguish the mechanistic basis underlying acute CFTR modulators","volume":"301","author":"Pyle","year":"2011","journal-title":"Am J Physiol Lung Cell Mol Physiol"},{"key":"2026030623190975300_bib28","doi-asserted-by":"crossref","first-page":"164","DOI":"10.1016\/j.cell.2011.11.023","article-title":"Requirements for efficient correction of \u0394F508 CFTR revealed by analyses of evolved sequences","volume":"148","author":"Mendoza","year":"2012","journal-title":"Cell"},{"key":"2026030623190975300_bib29","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1165\/rcmb.2006-0176OC","article-title":"Restoration of W1282X CFTR activity by enhanced expression","volume":"37","author":"Rowe","year":"2007","journal-title":"Am J Respir Cell Mol Biol"},{"key":"2026030623190975300_bib30","doi-asserted-by":"crossref","first-page":"1226","DOI":"10.1111\/j.1471-4159.2009.05871.x","article-title":"Aminoglycoside-induced histone deacetylation and hair cell death in the mouse cochlea","volume":"108","author":"Chen","year":"2009","journal-title":"J Neurochem"},{"key":"2026030623190975300_bib31","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.jpba.2012.11.014","article-title":"Development of generic immunoassay for the detection of a series of aminoglycosides with 6\u2032-OH group for the treatment of genetic diseases in biological samples","volume":"75","author":"Shalev","year":"2013","journal-title":"J Pharm Biomed Anal"},{"key":"2026030623190975300_bib32","doi-asserted-by":"crossref","first-page":"3735","DOI":"10.1016\/j.bmc.2010.03.060","article-title":"Repairing faulty genes by aminoglycosides: development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations","volume":"18","author":"Nudelman","year":"2010","journal-title":"Bioorg Med Chem"},{"key":"2026030623190975300_bib33","doi-asserted-by":"crossref","first-page":"18825","DOI":"10.1073\/pnas.0904709106","article-title":"Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770","volume":"106","author":"Van Goor","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"2026030623190975300_bib34","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa1105185","article-title":"A CFTR potentiator in patients with cystic fibrosis and the G551D mutation","volume":"365","author":"Ramsey","year":"2011","journal-title":"N Engl J Med"},{"key":"2026030623190975300_bib35","doi-asserted-by":"crossref","first-page":"2277","DOI":"10.2165\/11597020-000000000-00000","article-title":"Pharmacological considerations for the proper clinical use of aminoglycosides","volume":"71","author":"Pagkalis","year":"2011","journal-title":"Drugs"},{"key":"2026030623190975300_bib36","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1016\/j.jmb.2004.03.012","article-title":"Alpha-L-iduronidase premature stop codons and potential read-through in mucopolysaccharidosis type I patients","volume":"338","author":"Hein","year":"2004","journal-title":"J Mol Biol"},{"key":"2026030623190975300_bib37","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1093\/hmg\/10.3.291","article-title":"Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation","volume":"10","author":"Keeling","year":"2001","journal-title":"Hum Mol Genet"},{"key":"2026030623190975300_bib38","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2011.10.005","article-title":"The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse","volume":"105","author":"Wang","year":"2012","journal-title":"Mol Genet Metab"},{"key":"2026030623190975300_bib39","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1053\/ejpn.2000.0436","article-title":"Aminoglycoside-mediated suppression of nonsense mutations in late infantile neuronal ceroid lipofuscinosis","volume":"5","author":"Sleat","year":"2001","journal-title":"Eur J Paediatr Neurol"},{"key":"2026030623190975300_bib40","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1007\/s00109-010-0704-4","article-title":"Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse model","volume":"89","author":"Brendel","year":"2011","journal-title":"J Mol Med (Berl)"},{"key":"2026030623190975300_bib41","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1007\/s00439-007-0410-7","article-title":"In vitro and ex vivo suppression by aminoglycosides of PCDH15 nonsense mutations underlying type 1 Usher syndrome","volume":"122","author":"Rebibo-Sabbah","year":"2007","journal-title":"Hum Genet"},{"key":"2026030623190975300_bib42","doi-asserted-by":"crossref","first-page":"720","DOI":"10.1002\/ajh.21539","article-title":"Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients","volume":"84","author":"Salvatori","year":"2009","journal-title":"Am J Hematol"},{"key":"2026030623190975300_bib43","doi-asserted-by":"crossref","first-page":"e13659","DOI":"10.1371\/journal.pone.0013659","article-title":"The effect of gentamicin-induced readthrough on a novel premature termination codon of CD18 leukocyte adhesion deficiency patients","volume":"5","author":"Simon","year":"2010","journal-title":"PLoS One"},{"key":"2026030623190975300_bib44","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1002\/ana.22024","article-title":"Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy","volume":"67","author":"Malik","year":"2010","journal-title":"Ann Neurol"},{"key":"2026030623190975300_bib45","first-page":"15","article-title":"Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results","volume":"22","author":"Politano","year":"2003","journal-title":"Acta Myol"},{"key":"2026030623190975300_bib46","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1002\/ana.1023","article-title":"Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations","volume":"49","author":"Wagner","year":"2001","journal-title":"Ann Neurol"},{"key":"2026030623190975300_bib47","doi-asserted-by":"crossref","first-page":"700","DOI":"10.1038\/nrm3454","article-title":"NMD: a multifaceted response to premature translational termination","volume":"13","author":"Kervestin","year":"2012","journal-title":"Nat Rev Mol Cell Biol"},{"key":"2026030623190975300_bib48","doi-asserted-by":"crossref","first-page":"18843","DOI":"10.1073\/pnas.1105787108","article-title":"Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809","volume":"108","author":"Van Goor","year":"2011","journal-title":"Proc Natl Acad Sci USA"},{"key":"2026030623190975300_bib49","first-page":"Abstract 218","article-title":"Enhanced activity for translational readthrough and reduced toxicity demonstrated by the synthetic aminoglycoside NB54 [abstract]","volume":"44","author":"Rowe","year":"2009","journal-title":"Ped Pulmonol Suppl"},{"key":"2026030623190975300_bib50","doi-asserted-by":"crossref","first-page":"3585","DOI":"10.1073\/pnas.0813345106","article-title":"Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression","volume":"106","author":"Auld","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"2026030623190975300_bib51","doi-asserted-by":"crossref","first-page":"573","DOI":"10.1007\/s00109-006-0045-5","article-title":"Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model","volume":"84","author":"Du","year":"2006","journal-title":"J Mol Med (Berl)"},{"key":"2026030623190975300_bib52","doi-asserted-by":"crossref","first-page":"e1000155","DOI":"10.1371\/journal.pbio.1000155","article-title":"CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium","volume":"7","author":"Zhang","year":"2009","journal-title":"PLoS Biol"},{"key":"2026030623190975300_bib53","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1038\/ng0992-21","article-title":"Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis","volume":"2","author":"Johnson","year":"1992","journal-title":"Nat Genet"},{"key":"2026030623190975300_bib54","doi-asserted-by":"crossref","first-page":"1705","DOI":"10.1126\/science.7527588","article-title":"Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR","volume":"266","author":"Zhou","year":"1994","journal-title":"Science"},{"key":"2026030623190975300_bib55","doi-asserted-by":"crossref","first-page":"e60478","DOI":"10.1371\/journal.pone.0060478","article-title":"Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppression","volume":"8","author":"Keeling","year":"2013","journal-title":"PLoS ONE"}],"container-title":["American Journal of Respiratory Cell and Molecular Biology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/ajrcmb\/article-pdf\/50\/4\/805\/67114321\/ajrcmb_50_4_805.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/ajrcmb\/article-pdf\/50\/4\/805\/67114321\/ajrcmb_50_4_805.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T04:19:28Z","timestamp":1772857168000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/ajrcmb\/article\/50\/4\/805\/8492574"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,4,1]]},"references-count":55,"journal-issue":{"issue":"4","published-online":{"date-parts":[[2014,4,1]]},"published-print":{"date-parts":[[2014,4,1]]}},"URL":"https:\/\/doi.org\/10.1165\/rcmb.2013-0282oc","relation":{},"ISSN":["1044-1549","1535-4989"],"issn-type":[{"value":"1044-1549","type":"print"},{"value":"1535-4989","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2014,4]]},"published":{"date-parts":[[2014,4,1]]}}}