{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T12:11:15Z","timestamp":1777378275634,"version":"3.51.4"},"reference-count":42,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2022,6,1]],"date-time":"2022-06-01T00:00:00Z","timestamp":1654041600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,6,1]],"date-time":"2022-06-01T00:00:00Z","timestamp":1654041600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,4,11]],"date-time":"2022-04-11T00:00:00Z","timestamp":1649635200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000850","name":"Imperial College London NHLI","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000850","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010269","name":"Wellcome Trust","doi-asserted-by":"publisher","award":["110579\/Z\/15"],"award-info":[{"award-number":["110579\/Z\/15"]}],"id":[{"id":"10.13039\/100010269","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000272","name":"National Institute for Health and Care Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000735","name":"University of Cambridge","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000735","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2022,6]]},"DOI":"10.1016\/j.omtm.2022.04.002","type":"journal-article","created":{"date-parts":[[2022,4,14]],"date-time":"2022-04-14T11:26:20Z","timestamp":1649935580000},"page":"382-391","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"special_numbering":"C","title":["Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression"],"prefix":"10.1016","volume":"25","author":[{"given":"Helena","family":"Lund-Palau","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudia Ivette","family":"Juarez-Molina","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cuixiang","family":"Meng","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anushka","family":"Bhargava","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aikaterini","family":"Pilou","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kiran","family":"Aziz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nora","family":"Clarke","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Naoko","family":"Atsumi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ali","family":"Ashek","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael R.","family":"Wilson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masao","family":"Takata","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Simon","family":"Padley","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Deborah R.","family":"Gill","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephen C.","family":"Hyde","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cliff","family":"Morgan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3852-6764","authenticated-orcid":false,"given":"Eric W.F.W.","family":"Alton","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Uta","family":"Griesenbach","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2022.04.002_bib1","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.1038\/mt.2010.13","article-title":"Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes","volume":"18","author":"Mitomo","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.002_bib2","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1136\/thoraxjnl-2016-208406","article-title":"Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis","volume":"72","author":"Alton","year":"2017","journal-title":"Thorax"},{"key":"10.1016\/j.omtm.2022.04.002_bib3","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1006\/mthe.2002.0524","article-title":"The nasal epithelium as a factory for systemic protein delivery","volume":"5","author":"Griesenbach","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.002_bib4","doi-asserted-by":"crossref","first-page":"1123","DOI":"10.1056\/NEJM195806052582301","volume":"258","author":"Rosen","year":"1958","journal-title":"Pulm. Alveolar Proteinosis. N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib5","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1038\/s41572-019-0066-3","article-title":"Pulmonary alveolar proteinosis","volume":"5","author":"Trapnell","year":"2019","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.omtm.2022.04.002_bib6","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1164\/rccm.2109105","article-title":"Pulmonary alveolar proteinosis: progress in the first 44 years","volume":"166","author":"Seymour","year":"2002","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib7","doi-asserted-by":"crossref","first-page":"L650","DOI":"10.1152\/ajplung.1996.270.4.L650","article-title":"Surfactant granulocyte metabolism in transgenic mice after granulocyte macrophage-colony stimulating factor ablation","volume":"270","author":"Ikegami","year":"1996","journal-title":"Am. J. Physiol. Lung Cell Mol. Physiol."},{"key":"10.1016\/j.omtm.2022.04.002_bib8","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1172\/JCI118461","article-title":"Pulmonary epithelial cell expression of GM-CSF corrects the alveolar proteinosis in GM-CSF-deficient mice","volume":"97","author":"Huffman","year":"1996","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2022.04.002_bib9","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1146\/annurev.physiol.64.090601.113847","article-title":"GM-CSF regulates pulmonary surfactant homeostasis and alveolar macrophage-mediated innate host defense","volume":"64","author":"Trapnell","year":"2002","journal-title":"Annu. Rev. Physiol."},{"key":"10.1016\/j.omtm.2022.04.002_bib10","doi-asserted-by":"crossref","first-page":"752","DOI":"10.1164\/rccm.200708-1271OC","article-title":"Characteristics of a large cohort of patients with autoimmune pulmonary alveolar proteinosis in Japan","volume":"177","author":"Inoue","year":"2008","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib11","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1084\/jem.190.6.875","article-title":"Idiopathic pulmonary alveolar proteinosis as an autoimmune disease with neutralizing antibody against granulocyte\/macrophage colony-stimulating factor","volume":"190","author":"Kitamura","year":"1999","journal-title":"J.\u00a0Exp. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib12","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1165\/rcmb.2002-0023OC","article-title":"Autoantibodies against granulocyte macrophage colony-stimulating factor are diagnostic for pulmonary alveolar proteinosis","volume":"27","author":"Bonfield","year":"2002","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"10.1016\/j.omtm.2022.04.002_bib13","doi-asserted-by":"crossref","first-page":"2679","DOI":"10.1056\/NEJMc0904077","article-title":"Human GM-CSF autoantibodies and reproduction of pulmonary alveolar proteinosis","volume":"361","author":"Sakagami","year":"2009","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib14","doi-asserted-by":"crossref","first-page":"1089","DOI":"10.1182\/blood-2003-05-1565","article-title":"High affinity autoantibodies specifically eliminate granulocyte-macrophage colony stimulating factor activity in the lung of patients with idiopathic pulmonary alveolar proteinosis","volume":"103","author":"Uchida","year":"2004","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2022.04.002_bib15","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1016\/0002-9343(68)90062-4","article-title":"Lung lavage (alveolar washing) in alveolar proteinosis","volume":"44","author":"Wasserman","year":"1968","journal-title":"Am. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib16","doi-asserted-by":"crossref","first-page":"526","DOI":"10.1183\/09031936.04.00102704","article-title":"Long-term durable benefit after whole lung lavage in pulmonary alveolar proteinosis","volume":"23","author":"Beccaria","year":"2004","journal-title":"Eur. Respir. J."},{"key":"10.1016\/j.omtm.2022.04.002_bib17","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1186\/s13023-016-0497-9","article-title":"Whole lung lavage therapy for pulmonary alveolar proteinosis: a global survey of current practices and procedures","volume":"11","author":"Campo","year":"2016","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2022.04.002_bib18","doi-asserted-by":"crossref","first-page":"2714","DOI":"10.4103\/0366-6999.167295","article-title":"Whole lung lavage treatment of Chinese patients with autoimmune pulmonary alveolar proteinosis: a retrospective long-term follow-up study","volume":"128","author":"Zhao","year":"2015","journal-title":"Chin. Med. J. (Engl)."},{"key":"10.1016\/j.omtm.2022.04.002_bib19","doi-asserted-by":"crossref","first-page":"1924","DOI":"10.1056\/NEJM199612193352513","article-title":"Efficacy of granulocyte-macrophage colony-stimulating factor in acquired alveolar proteinosis","volume":"335","author":"Seymour","year":"1996","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib20","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1164\/ajrccm.163.2.2003146","article-title":"Therapeutic efficacy of granulocyte-macrophage colony-stimulating factor in patients with idiopathic acquired alveolar proteinosis","volume":"163","author":"Seymour","year":"2001","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib21","doi-asserted-by":"crossref","first-page":"1142","DOI":"10.1164\/rccm.200406-716OC","article-title":"Granulocyte-macrophage colony-stimulating factor and lung immunity in pulmonary alveolar proteinosis","volume":"171","author":"Tazawa","year":"2005","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib22","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1378\/chest.130.1.227","article-title":"An open-label trial of granulocyte macrophage colony stimulating factor therapy for moderate symptomatic pulmonary alveolar proteinosis","volume":"130","author":"Venkateshiah","year":"2006","journal-title":"Chest"},{"key":"10.1016\/j.omtm.2022.04.002_bib23","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1164\/rccm.200906-0978OC","article-title":"Inhaled granulocyte\/macrophage-colony stimulating factor as therapy for pulmonary alveolar proteinosis","volume":"181","author":"Tazawa","year":"2010","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib24","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1378\/chest.13-0603","article-title":"Duration of benefit in patients with autoimmune pulmonary alveolar proteinosis after inhaled granulocyte-macrophage colony-stimulating factor therapy","volume":"145","author":"Tazawa","year":"2014","journal-title":"Chest"},{"key":"10.1016\/j.omtm.2022.04.002_bib25","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1056\/NEJMoa1816216","article-title":"Inhaled GM-CSF for pulmonary alveolar proteinosis","volume":"381","author":"Tazawa","year":"2019","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib26","doi-asserted-by":"crossref","first-page":"1635","DOI":"10.1056\/NEJMoa1913590","article-title":"Inhaled molgramostim therapy in autoimmune pulmonary alveolar proteinosis","volume":"383","author":"Trapnell","year":"2020","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib27","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s12931-018-0862-4","article-title":"Better approach for autoimmune pulmonary alveolar proteinosis treatment: inhaled or subcutaneous granulocyte-macrophage colony-stimulating factor: a meta-analyses","volume":"19","author":"Sheng","year":"2018","journal-title":"Respir. Res."},{"key":"10.1016\/j.omtm.2022.04.002_bib28","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1126\/science.8171324","article-title":"Involvement of granulocyte-macrophage colony-stimulating factor in pulmonary homeostasis","volume":"264","author":"Dranoff","year":"1994","journal-title":"Science"},{"key":"10.1016\/j.omtm.2022.04.002_bib29","doi-asserted-by":"crossref","first-page":"L556","DOI":"10.1152\/ajplung.1999.276.4.L556","article-title":"Aerosolized GM-CSF ameliorates pulmonary alveolar proteinosis in GM-CSF-deficient mice","volume":"276","author":"Reed","year":"1999","journal-title":"Am. J. Physiol. Cell. Mol. Physiol."},{"key":"10.1016\/j.omtm.2022.04.002_bib30","doi-asserted-by":"crossref","first-page":"10684","DOI":"10.1128\/JVI.00227-08","article-title":"Lentivirus vector can Be readministered to nasal epithelia without blocking immune responses","volume":"82","author":"Sinn","year":"2008","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2022.04.002_bib31","doi-asserted-by":"crossref","first-page":"846","DOI":"10.1164\/rccm.201206-1056OC","article-title":"Assessment of F\/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy","volume":"186","author":"Griesenbach","year":"2012","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib32","doi-asserted-by":"crossref","first-page":"2101","DOI":"10.1089\/hum.1998.9.14-2101","article-title":"Adenovirus-mediated granulocyte-macrophage colony-stimulating factor improves lung pathology of pulmonary alveolar proteinosis in granulocyte-macrophage colony-stimulating factor-deficient mice","volume":"2109","author":"Zsengeller","year":"1998","journal-title":"Hum. Gene. Ther."},{"key":"10.1016\/j.omtm.2022.04.002_bib33","doi-asserted-by":"crossref","first-page":"2004","DOI":"10.1128\/jvi.69.4.2004-2015.1995","article-title":"Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses","volume":"69","author":"Yang","year":"1995","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2022.04.002_bib34","doi-asserted-by":"crossref","first-page":"1998","DOI":"10.1016\/S0021-9258(18)71855-3","article-title":"Purification and properties of colony-stimulating from mouse lung-conditioned medium","volume":"252","author":"Burgess","year":"1977","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2022.04.002_bib35","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1056\/NEJMoa062505","article-title":"GM-CSF autoantibodies and neutrophil dysfunction in pulmonary alveolar proteinosis","volume":"356","author":"Uchida","year":"2007","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib36","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1016\/0092-8674(87)90136-X","article-title":"Transgenic mice expressing a hemopoietic growth factor gene (GM-CSF) develop accumulations of macrophages, blindness, and a fatal syndrome of tissue damage","volume":"51","author":"Lang","year":"1987","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2022.04.002_bib37","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1002\/j.1460-2075.1989.tb03396.x","article-title":"A lethal myeloproliferative syndrome in mice transplanted with bone marrow cells infected with a retrovirus expressing granulocyte-macrophage colony stimulating factor","volume":"8","author":"Johnson","year":"1989","journal-title":"EMBO J."},{"key":"10.1016\/j.omtm.2022.04.002_bib38","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1002\/ajh.2830340117","article-title":"Recurrent spleen enlargement during cyclic granulocyte-macrophage colony-stimulating factor therapy for myelodysplastic syndrome","volume":"34","author":"Delmer","year":"1990","journal-title":"Am. Jounral Hematol."},{"key":"10.1016\/j.omtm.2022.04.002_bib39","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1016\/0145-2126(93)90012-A","article-title":"Fatal spleen rupture during induction chemotherapy with RH GM-CSF priming for acute monocytic leukemia. Clinical case report and in\u00a0vitro studies","volume":"17","author":"Zimmer","year":"1993","journal-title":"Leuk. Res."},{"key":"10.1016\/j.omtm.2022.04.002_bib40","doi-asserted-by":"crossref","first-page":"470","DOI":"10.4049\/jimmunol.1502248","article-title":"Autoantibody-mediated pulmonary alveolar proteinosis in rasgrp1-deficient mice","volume":"197","author":"Feretti","year":"2016","journal-title":"J.\u00a0Immunol."},{"key":"10.1016\/j.omtm.2022.04.002_bib41","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1164\/rccm.201001-0008OC","article-title":"Patient-derived granulocyte\/macrophage colony-stimulating factor autoantibodies reproduce pulmonary alveolar proteinosis in nonhuman primates","volume":"182","author":"Sakagami","year":"2010","journal-title":"Am. J. Respir. Crit. Care Med."},{"key":"10.1016\/j.omtm.2022.04.002_bib42","doi-asserted-by":"crossref","first-page":"1883","DOI":"10.1002\/eji.201847580","article-title":"Prophylactic treatment against GM-CSF, but not IL-17, abolishes relapses in a chronic murine model of multiple sclerosis","volume":"48","author":"Uyttenhove","year":"2018","journal-title":"Eur. J. Immunol."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122000493?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122000493?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:30:15Z","timestamp":1762421415000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050122000493"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6]]},"references-count":42,"alternative-id":["S2329050122000493"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2022.04.002","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2022,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2022.04.002","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Authors.","name":"copyright","label":"Copyright"}]}}