{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,19]],"date-time":"2026-03-19T13:58:29Z","timestamp":1773928709863,"version":"3.50.1"},"reference-count":41,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T00:00:00Z","timestamp":1762819200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100009252","name":"Science for Life Laboratory","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100009252","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100015701","name":"Uppsala Multidisciplinary Center for Advanced Computational Science","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100015701","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100008385","name":"Cystic Fibrosis Research Foundation","doi-asserted-by":"publisher","award":["FFC#05_2015"],"award-info":[{"award-number":["FFC#05_2015"]}],"id":[{"id":"10.13039\/501100008385","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003100","name":"F\u00e9d\u00e9ration Fran\u00e7aise de Cardiologie","doi-asserted-by":"publisher","award":["05_2019"],"award-info":[{"award-number":["05_2019"]}],"id":[{"id":"10.13039\/501100003100","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","cysticfibrosisjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Cystic Fibrosis"],"published-print":{"date-parts":[[2026,1]]},"DOI":"10.1016\/j.jcf.2025.11.006","type":"journal-article","created":{"date-parts":[[2025,11,20]],"date-time":"2025-11-20T01:43:40Z","timestamp":1763603020000},"page":"118-126","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues"],"prefix":"10.1016","volume":"25","author":[{"given":"Valeria","family":"Rimoldi","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0972-610X","authenticated-orcid":false,"given":"Giulia","family":"Sold\u00e0","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anita","family":"Capalbo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elena","family":"Saba","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Valentina","family":"Giannone","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9740-1171","authenticated-orcid":false,"given":"Valeria","family":"Capurro","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1355-8252","authenticated-orcid":false,"given":"Laura","family":"Lentini","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raffaella","family":"Melfi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Massimo","family":"Lazzeri","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3172-6528","authenticated-orcid":false,"given":"Luigi","family":"Porcaro","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Manuela","family":"Seia","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1916-2440","authenticated-orcid":false,"given":"Massimo","family":"Aureli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5161-1720","authenticated-orcid":false,"given":"Nicoletta","family":"Pedemonte","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stefano","family":"Duga","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christian","family":"Orrenius","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5351-0619","authenticated-orcid":false,"given":"Rosanna","family":"Asselta","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1733-7561","authenticated-orcid":false,"given":"Letizia","family":"Straniero","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.jcf.2025.11.006_bib0001","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a009472","article-title":"The cystic fibrosis gene: a molecular genetic perspective","volume":"3","author":"Tsui","year":"2013","journal-title":"Cold Spring Harb Perspect Med"},{"key":"10.1016\/j.jcf.2025.11.006_bib0002","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1038\/ng0293-151","article-title":"Genetic basis of variable exon 9 skipping in cystic fibrosis transmembrane conductance regulator mRNA","volume":"3","author":"Chu","year":"1993","journal-title":"Nat Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0003","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1093\/hmg\/2.3.225","article-title":"Expression of an abundant alternatively spliced form of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is not associated with a cAMP-activated chloride conductance","volume":"2","author":"Strong","year":"1993","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0004","doi-asserted-by":"crossref","first-page":"ugae017","DOI":"10.1093\/narmme\/ugae017","article-title":"Systematic deletion of symmetrical CFTR exons reveals new therapeutic targets for exon skipping antisense oligonucleotides","volume":"1","author":"Pena-Rasgado","year":"2024","journal-title":"NAR Molecular Medicine"},{"key":"10.1016\/j.jcf.2025.11.006_bib0005","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1016\/j.jcf.2021.03.015","article-title":"A survey of the prevalence, management and outcome of infants with an inconclusive diagnosis following newborn bloodspot screening for cystic fibrosis (CRMS\/CFSPID) in six Italian centres","volume":"20","author":"Terlizzi","year":"2021","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2025.11.006_bib0006","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a009480","article-title":"Assessing the Disease-Liability of Mutations in CFTR","volume":"2","author":"Ferec","year":"2012","journal-title":"Cold Spring Harb Perspect Med"},{"key":"10.1016\/j.jcf.2025.11.006_bib0007","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1016\/S2213-2600(12)70057-7","article-title":"A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect","volume":"1","author":"Boyle","year":"2013","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.jcf.2025.11.006_bib0008","doi-asserted-by":"crossref","first-page":"S46","DOI":"10.1016\/j.jcf.2017.10.017","article-title":"Translational research to enable personalized treatment of cystic fibrosis","volume":"17","author":"Hagemeijer","year":"2018","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2025.11.006_bib0009","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1038\/s41434-022-00347-0","article-title":"Splicing mutations in the CFTR gene as therapeutic targets","volume":"29","author":"Deletang","year":"2022","journal-title":"Gene Ther"},{"key":"10.1016\/j.jcf.2025.11.006_bib0010","doi-asserted-by":"crossref","DOI":"10.3390\/ijms241914617","article-title":"Development of Engineered-U1 snRNA Therapies: current Status","volume":"24","author":"Gon\u00e7alves","year":"2023","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.jcf.2025.11.006_bib0011","doi-asserted-by":"crossref","first-page":"10","DOI":"10.14348\/molcells.2023.2172","article-title":"Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: recent Developments and Perspectives","volume":"46","author":"Kim","year":"2023","journal-title":"Mol Cells"},{"key":"10.1016\/j.jcf.2025.11.006_bib0012","first-page":"1317","article-title":"U7 snRNA, a Small RNA with a Big Impact in Gene Therapy","volume":"32","author":"Lesman","year":"2021","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jcf.2025.11.006_bib0013","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1089\/hum.2020.286","article-title":"Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping","volume":"32","author":"Gushchina","year":"2021","journal-title":"Hum Gene Ther"},{"key":"10.1016\/j.jcf.2025.11.006_bib0014","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1093\/hmg\/ddh046","article-title":"Rescue of a human mRNA splicing defect by the plant cytokinin kinetin","volume":"13","author":"Slaugenhaupt","year":"2004","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0015","doi-asserted-by":"crossref","first-page":"614","DOI":"10.1038\/ejhg.2009.212","article-title":"Modulation of aberrant NF1 pre-mRNA splicing by kinetin treatment","volume":"18","author":"Pros","year":"2010","journal-title":"Eur J Hum Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0016","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1126\/science.1250127","article-title":"Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy","volume":"345","author":"Naryshkin","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.jcf.2025.11.006_bib0017","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1007\/s00109-006-0137-2","article-title":"Therapeutic potential and mechanism of kinetin as a treatment for the human splicing disease familial dysautonomia","volume":"85","author":"Hims","year":"2007","journal-title":"J Mol Med (Berl)"},{"key":"10.1016\/j.jcf.2025.11.006_bib0018","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1203\/PDR.0b013e31822e1825","article-title":"Kinetin improves IKBKAP mRNA splicing in patients with familial dysautonomia","volume":"70","author":"Axelrod","year":"2011","journal-title":"Pediatr Res"},{"key":"10.1016\/j.jcf.2025.11.006_bib0019","doi-asserted-by":"crossref","first-page":"2764","DOI":"10.1073\/pnas.1415525112","article-title":"Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia","volume":"112","author":"Yoshida","year":"2015","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.jcf.2025.11.006_bib0020","doi-asserted-by":"crossref","first-page":"3332","DOI":"10.1038\/s41467-021-23663-2","article-title":"A deep learning approach to identify gene targets of a therapeutic for human splicing disorders","volume":"12","author":"Gao","year":"2021","journal-title":"Nat Commun"},{"key":"10.1016\/j.jcf.2025.11.006_bib0021","doi-asserted-by":"crossref","first-page":"614","DOI":"10.1038\/ejhg.2009.212","article-title":"Modulation of aberrant NF1 pre-mRNA splicing by kinetin treatment","volume":"18","author":"Pros","year":"2010","journal-title":"Eur J Hum Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0022","doi-asserted-by":"crossref","first-page":"1291","DOI":"10.1093\/nar\/gkt987","article-title":"Functional characterization of the common c.-32-13T>G mutation of GAA gene: identification of potential therapeutic agents","volume":"42","author":"Dardis","year":"2014","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.jcf.2025.11.006_bib0023","doi-asserted-by":"crossref","first-page":"1191","DOI":"10.2741\/3670","article-title":"Splicing of the Survival Motor Neuron genes and implications for treatment of SMA","volume":"15","author":"Bebee","year":"2010","journal-title":"Front Biosci (Landmark Ed)"},{"key":"10.1016\/j.jcf.2025.11.006_bib0024","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1002\/humu.22010","article-title":"Genome-wide analysis of familial dysautonomia and kinetin target genes with patient olfactory ecto-mesenchymal stem cells","volume":"33","author":"Boone","year":"2012","journal-title":"Hum Mutat"},{"key":"10.1016\/j.jcf.2025.11.006_bib0025","doi-asserted-by":"crossref","first-page":"e148","DOI":"10.1093\/nar\/gkl967","article-title":"A bichromatic fluorescent reporter for cell-based screens of alternative splicing","volume":"34","author":"Orengo","year":"2006","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.jcf.2025.11.006_bib0026","doi-asserted-by":"crossref","first-page":"21041","DOI":"10.1074\/jbc.M910165199","article-title":"Splicing factors induce cystic fibrosis transmembrane regulator exon 9 skipping through a nonevolutionary conserved intronic element","volume":"275","author":"Pagani","year":"2000","journal-title":"J Biol Chem"},{"key":"10.1016\/j.jcf.2025.11.006_bib0027","doi-asserted-by":"crossref","first-page":"2143","DOI":"10.1002\/humu.24116","article-title":"Rescue of common exon-skipping mutations in cystic fibrosis with modified U1 snRNAs","volume":"41","author":"Doneg\u00e0","year":"2020","journal-title":"Hum Mutat"},{"key":"10.1016\/j.jcf.2025.11.006_bib0028","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1186\/s13058-017-0919-1","article-title":"Nanopore sequencing of full-length BRCA1 mRNA transcripts reveals co-occurrence of known exon skipping events","volume":"19","author":"de Jong","year":"2017","journal-title":"Breast Cancer Res"},{"key":"10.1016\/j.jcf.2025.11.006_bib0029","doi-asserted-by":"crossref","first-page":"833","DOI":"10.1136\/jmg.30.10.833","article-title":"CFTR transcripts are undetectable in lymphocytes and respiratory epithelial cells of a CF patient homozygous for the nonsense mutation R553X","volume":"30","author":"Will","year":"1993","journal-title":"J Med Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0030","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1016\/j.jcf.2021.09.020","article-title":"Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids","volume":"21","author":"de Poel","year":"2022","journal-title":"Journal of Cystic Fibrosis"},{"key":"10.1016\/j.jcf.2025.11.006_bib0031","doi-asserted-by":"crossref","first-page":"1249","DOI":"10.1002\/humu.22624","article-title":"Experimental Assessment of Splicing Variants Using Expression Minigenes and Comparison with In Silico Predictions","volume":"35","author":"Sharma","year":"2014","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.jcf.2025.11.006_bib0032","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1007\/s00109-006-0137-2","article-title":"Therapeutic potential and mechanism of kinetin as a treatment for the human splicing disease familial dysautonomia","volume":"85","author":"Hims","year":"2007","journal-title":"J Mol Med (Berl)"},{"key":"10.1016\/j.jcf.2025.11.006_bib0033","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/j.ejpb.2020.09.002","article-title":"An in vitro model for assessing drug transport in cystic fibrosis treatment: characterisation of the CuFi-1 cell line","volume":"156","author":"Sheikh","year":"2020","journal-title":"Eur J Pharm Biopharm"},{"key":"10.1016\/j.jcf.2025.11.006_bib0034","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1016\/j.mcp.2004.03.005","article-title":"A comparison of 14 antibodies for the biochemical detection of the cystic fibrosis transmembrane conductance regulator protein","volume":"18","author":"Farinha","year":"2004","journal-title":"Mol Cell Probes"},{"key":"10.1016\/j.jcf.2025.11.006_bib0035","first-page":"87","article-title":"The molecular basis of partial penetrance of splicing mutations in cystic fibrosis","volume":"60","author":"Rave-Harel","year":"1997","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.jcf.2025.11.006_bib0036","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1165\/rcmb.2001-0004OC","article-title":"Five percent of normal cystic fibrosis transmembrane conductance regulator mRNA ameliorates the severity of pulmonary disease in cystic fibrosis","volume":"27","author":"Ramalho","year":"2002","journal-title":"Am J Respir Cell Mol Biol"},{"key":"10.1016\/j.jcf.2025.11.006_bib0037","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1165\/rcmb.2012-0371OC","article-title":"Fine characterization of the recurrent c.1584+18672A>G deep-intronic mutation in the cystic fibrosis transmembrane conductance regulator gene","volume":"48","author":"Costantino","year":"2013","journal-title":"Am J Respir Cell Mol Biol"},{"key":"10.1016\/j.jcf.2025.11.006_bib0038","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1093\/molehr\/gah214","article-title":"Gender-sensitive association of CFTR gene mutations and 5T allele emerging from a large survey on infertility","volume":"11","author":"Morea","year":"2005","journal-title":"Mol Hum Reprod"},{"key":"10.1016\/j.jcf.2025.11.006_bib0039","doi-asserted-by":"crossref","first-page":"1165","DOI":"10.1002\/humu.24250","article-title":"Elucidating clinical phenotypic variability associated with the polyT tract and TG repeats in CFTR","volume":"42","author":"Nykamp","year":"2021","journal-title":"Hum Mutat"},{"key":"10.1016\/j.jcf.2025.11.006_bib0040","doi-asserted-by":"crossref","first-page":"eadt9695","DOI":"10.1126\/sciadv.adt9695","article-title":"Invention of an oral medication for cardiac Fabry disease caused by RNA mis-splicing","volume":"11","author":"Awaya","year":"2025","journal-title":"Sci. Adv."},{"key":"10.1016\/j.jcf.2025.11.006_bib0041","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1016\/j.ajhg.2019.02.009","article-title":"ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia","volume":"104","author":"Morini","year":"2019","journal-title":"Am J Hum Genet"}],"container-title":["Journal of Cystic Fibrosis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199325025020?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199325025020?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,19]],"date-time":"2026-03-19T10:44:16Z","timestamp":1773917056000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1569199325025020"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":41,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["S1569199325025020"],"URL":"https:\/\/doi.org\/10.1016\/j.jcf.2025.11.006","relation":{},"ISSN":["1569-1993"],"issn-type":[{"value":"1569-1993","type":"print"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues","name":"articletitle","label":"Article Title"},{"value":"Journal of Cystic Fibrosis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jcf.2025.11.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.","name":"copyright","label":"Copyright"}]}}