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Pathogenic variants in CCDC40 are associated with a more severe reduction of lung function compared to most other PCD types. Currently, no therapies correcting the underlying disease mechanism are available. Here, we investigate the efficacy of lipidoid nanoparticle-formulated mRNA encoding human CCDC40 (LNP-CCDC40-mRNA) as a corrective measure for structural and functional defects in vitro (human cells) and in vivo (zebrafish). Human nasal respiratory epithelial cells cultured at an air-liquid interface from 5 CCDC40-deficient individuals and a newly generated vertebrate animal model (ccdc40\u2212\/\u2212 zebrafish) were treated with LNP-CCDC40-mRNA. CCDC40-deficient cells were analyzed by high-speed video microscopy and immunofluorescence microscopy. ccdc40\u2212\/\u2212 zebrafish olfactory pit cilia were analyzed by high-speed video microscopy and fluid flow assays. Topical application of exogenous LNP-CCDC40-mRNA to CCDC40-deficient cells results in endogenous CCDC40 expression (10%-74% of ciliated cells), enabling axonemal integration of CCDC40-associated proteins (CCDC39, GAS8\/DRC4, DNALI1). Consistently, ciliary beat frequencies were significantly increased in treated CCDC40-deficient cells and were comparable to those of healthy control cells. Further, we showed improved ciliary transport of fluorescent particles. Injection or topical application of human LNP-CCDC40-mRNA to ccdc40\u2212\/\u2212 zebrafish significantly increased ciliary motility and established directional flow in olfactory pits. We provide structural and functional evidence in vitro and in vivo for the biological efficacy of LNP-CCDC40-mRNA in CCDC40-deficient respiratory cells and zebrafish. Based on our results, an in vivo human study (Phase 1 trial) is planned in individuals with pathogenic variants in CCDC40.<\/jats:p>","DOI":"10.1093\/ajrcmb\/aanag034","type":"journal-article","created":{"date-parts":[[2026,3,26]],"date-time":"2026-03-26T12:46:19Z","timestamp":1774529179000},"page":"1237-1250","source":"Crossref","is-referenced-by-count":1,"title":["mRNA therapy improves the composition and motility in\n                    <i>CCDC40-<\/i>\n                    deficient cilia in vitro and in vivo"],"prefix":"10.1093","volume":"74","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4092-0664","authenticated-orcid":false,"given":"Kai","family":"Wohlgemuth","sequence":"first","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4891-4448","authenticated-orcid":false,"given":"Margarida","family":"Rasteiro","sequence":"additional","affiliation":[{"name":"iNOVA4Health, NOVA Medical School\/Faculdade de Ci\u00eancias M\u00e9dicas, NMS|FCM, Universidade Nova de Lisboa , Lisboa,","place":["Portugal"]},{"name":"Instituto de Medicina Molecular Jo\u00e3o Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa , Lisbon,","place":["Portugal"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manish","family":"Aneja","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6718-572X","authenticated-orcid":false,"given":"Catarina","family":"Bota","sequence":"additional","affiliation":[{"name":"Instituto de Medicina Molecular Jo\u00e3o Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa , Lisbon,","place":["Portugal"]},{"name":"Department of Animal Biology, Faculty of Sciences, University of Lisbon , Lisboa,","place":["Portugal"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8303-100X","authenticated-orcid":false,"given":"Sandra","family":"Cindric","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefanie","family":"Freischem","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sebastian","family":"George","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gizem","family":"G\u00fcnes G\u00fcnsel","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seun","family":"Ishola","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-4974-5540","authenticated-orcid":false,"given":"Julia","family":"Koenig","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5555-6941","authenticated-orcid":false,"given":"Rebekka","family":"Kubisch-Dohmen","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-6891-1968","authenticated-orcid":false,"given":"Thomas","family":"Langenickel","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1534-2105","authenticated-orcid":false,"given":"Niki Tomas","family":"Loges","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-1215-7685","authenticated-orcid":false,"given":"Miguel","family":"Lopes","sequence":"additional","affiliation":[{"name":"Department of Animal Biology, Faculty of Sciences, University of Lisbon , Lisboa,","place":["Portugal"]},{"name":"Department of Life Sciences, NOVA School of Science and Technology , Caparica,","place":["Portugal"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-8798-5257","authenticated-orcid":false,"given":"Verena","family":"Mummert","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4355-353X","authenticated-orcid":false,"given":"Heike","family":"Olbrich","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8202-3809","authenticated-orcid":false,"given":"Petra","family":"Pennekamp","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , 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Caparica,","place":["Portugal"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1571-2647","authenticated-orcid":false,"given":"Johanna","family":"Raidt","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9050-5675","authenticated-orcid":false,"given":"Carsten","family":"Rudolph","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adrian","family":"Dori\u00dfen ter Steege","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Drishti","family":"Valecha","sequence":"additional","affiliation":[{"name":"Ethris GmbH , Planegg,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6733-6356","authenticated-orcid":false,"given":"Susana S","family":"Lopes","sequence":"additional","affiliation":[{"name":"cE3c - Center for Ecology, Evolution and Environmental Changes & CHANGE - Global Change and Sustainability, Department of Animal Biology, Faculty of Sciences, University of Lisbon , Lisboa,","place":["Portugal"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0282-6765","authenticated-orcid":false,"given":"Heymut","family":"Omran","sequence":"additional","affiliation":[{"name":"Department of General Pediatrics, University Children\u2019s Hospital Muenster , Muenster,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2026,3,30]]},"reference":[{"key":"2026082703534792200_aanag034-B1","doi-asserted-by":"publisher","first-page":"459","DOI":"10.1016\/S2213-2600(21)00453-7","article-title":"The global prevalence and ethnic heterogeneity of primary ciliary dyskinesia gene variants: a genetic database analysis","volume":"10","author":"Hannah","year":"2022","journal-title":"Lancet Respir Med"},{"key":"2026082703534792200_aanag034-B2","doi-asserted-by":"publisher","first-page":"104171","DOI":"10.1016\/j.lpm.2023.104171","article-title":"Primary ciliary dyskinesia","volume":"52","author":"Raidt","year":"2023","journal-title":"Presse Med"},{"key":"2026082703534792200_aanag034-B3","doi-asserted-by":"publisher","first-page":"1264","DOI":"10.1183\/09031936.00176608","article-title":"Primary ciliary dyskinesia: a consensus statement on diagnostic and treatment approaches in children","volume":"34","author":"Barbato","year":"2009","journal-title":"Eur Respir J"},{"key":"2026082703534792200_aanag034-B4","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1038\/s41572-020-0209-6","article-title":"Motile ciliopathies","volume":"6","author":"Wallmeier","year":"2020","journal-title":"Nat Rev Dis Primers"},{"key":"2026082703534792200_aanag034-B5","doi-asserted-by":"publisher","first-page":"202","DOI":"10.1016\/S2213-2600(19)30374-1","article-title":"Primary ciliary dyskinesia in the genomics age","volume":"8","author":"Lucas","year":"2020","journal-title":"Lancet Respir Med"},{"key":"2026082703534792200_aanag034-B6","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1016\/j.semcdb.2020.11.007","article-title":"Motile cilia and airway disease","volume":"110","author":"Legendre","year":"2021","journal-title":"Semin Cell Dev 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Mutat"},{"key":"2026082703534792200_aanag034-B10","doi-asserted-by":"publisher","first-page":"190","DOI":"10.1164\/rccm.201803-0548OC","article-title":"Primary ciliary dyskinesia: longitudinal study of lung disease by ultrastructure defect and genotype","volume":"199","author":"Davis","year":"2019","journal-title":"Am J Respir Crit Care Med"},{"key":"2026082703534792200_aanag034-B11","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.01769-2023","article-title":"Analyses of 1236 genotyped primary ciliary dyskinesia individuals identify regional clusters of distinct DNA variants and significant genotype-phenotype correlations","volume":"64","author":"Raidt","year":"2024","journal-title":"Eur Respir J"},{"key":"2026082703534792200_aanag034-B12","doi-asserted-by":"publisher","first-page":"eadp5173","DOI":"10.1126\/scitranslmed.adp5173","article-title":"Undocking of an extensive ciliary network induces proteostasis and cell fate switching resulting in severe primary ciliary 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