{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T19:21:55Z","timestamp":1792092115827,"version":"4.4.0"},"reference-count":54,"publisher":"The Company of Biologists","issue":"20","license":[{"start":{"date-parts":[[2025,10,27]],"date-time":"2025-10-27T00:00:00Z","timestamp":1761523200000},"content-version":"vor","delay-in-days":12,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100007890","name":"University of North Carolina at Chapel Hill","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100007890","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,10,15]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Primary ciliary dyskinesia (PCD) is a rare, genetically heterogenous disorder resulting from dysfunctional motile cilia that is characterized by chronic, progressive lung disease with currently no corrective therapies available. Here, we test the efficacy of selective organ targeting lipid nanoparticles (SORT-LNPs) that were optimized for potency and delivery to respiratory cells containing an mRNA encoding an axonemal protein to rescue ciliary activity in a murine culture model of PCD. Utilizing murine nasopharyngeal epithelial cell (mNPEC) cultures isolated from a conditional Dnai1 knockout mouse model of the known human PCD-associated gene DNAI1 homolog, we tested if SORT-LNPs containing an optimized Dnai1 mRNA could rescue ciliary activity. Treatment of differentiating and well-differentiated Dnai1 knockout mNPECs with SORT-LNP-Dnai1 mRNA led to a dose-dependent increase in levels of DNAI1 protein and incorporation into ciliary axonemes, resulting in rescued ciliary activity with normal ciliary beat frequency that persisted for over 3 weeks. These data support further clinical development of an mRNA-based therapeutic with LNP-mediated delivery as a treatment for individuals with PCD with disease-causing DNAI1 mutations.<\/jats:p>","DOI":"10.1242\/jcs.264068","type":"journal-article","created":{"date-parts":[[2025,9,18]],"date-time":"2025-09-18T08:11:10Z","timestamp":1758183070000},"source":"Crossref","is-referenced-by-count":7,"title":["Lipid nanoparticle-encapsulated\n                    <i>Dnai1<\/i>\n                    mRNA rescues ciliary activity in primary ciliary dyskinesia mouse cell models"],"prefix":"10.1242","volume":"138","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3693-7917","authenticated-orcid":false,"given":"Amanda J.","family":"Smith","sequence":"first","affiliation":[{"name":"Marsico Lung Institute\/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill 1 , Chapel Hill, NC 27599 ,","place":["USA"]},{"name":"Presently ReCode Therapeutics 2 , Durham, NC 27701"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick R.","family":"Sears","sequence":"additional","affiliation":[{"name":"Marsico Lung Institute\/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill 1 , Chapel Hill, NC 27599 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-0238-737X","authenticated-orcid":false,"given":"Mirko","family":"Hennig","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rumpa B.","family":"Bhattacharjee","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weining","family":"Yin","sequence":"additional","affiliation":[{"name":"Marsico Lung Institute\/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill 1 , Chapel Hill, NC 27599 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hannah","family":"Golliher","sequence":"additional","affiliation":[{"name":"Marsico Lung Institute\/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill 1 , Chapel Hill, NC 27599 ,","place":["USA"]},{"name":"Department of Molecular Genetics and Microbiology, University of Florida 4 , Gainesville, FL 32610 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-3443-6869","authenticated-orcid":false,"given":"Daniella","family":"Ishimaru","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. Noelle","family":"Lombana","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David J.","family":"Lockhart","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brandon A.","family":"Wustman","sequence":"additional","affiliation":[{"name":"ReCode Therapeutics 3 , Menlo Park, CA 94025 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7558-6603","authenticated-orcid":false,"given":"Lawrence E.","family":"Ostrowski","sequence":"additional","affiliation":[{"name":"Marsico Lung Institute\/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill 1 , Chapel Hill, NC 27599 ,","place":["USA"]},{"name":"Department of Pediatrics, University of North Carolina at Chapel Hill 5 , Chapel Hill, NC 27599 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"237","published-online":{"date-parts":[[2025,10,27]]},"reference":[{"key":"2025102705193067600_JCS264068C1","doi-asserted-by":"publisher","first-page":"195","DOI":"10.2144\/000112531","article-title":"Murine nasal septa for respiratory epithelial air-liquid interface cultures","volume":"43","author":"Antunes","year":"2007","journal-title":"BioTechniques"},{"key":"2025102705193067600_JCS264068C55","doi-asserted-by":"publisher","first-page":"6085","DOI":"10.1128\/JVI.73.7.6085-6088.1999","article-title":"Transduction of well-differentiated airway epithelium by recombinant adeno-associated virus is limited by vector entry","volume":"73","author":"Bals","year":"1999","journal-title":"J. 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