{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T00:08:45Z","timestamp":1790208525220,"version":"4.1.0"},"reference-count":40,"publisher":"SAGE Publications","issue":"15-16","license":[{"start":{"date-parts":[[2025,8,1]],"date-time":"2025-08-01T00:00:00Z","timestamp":1754006400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:p>\n                    Cystic fibrosis (CF) is caused by mutations in the\n                    <jats:italic toggle=\"yes\">cystic fibrosis transmembrane conductance regulator<\/jats:italic>\n                    (\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    ). While gene therapy holds promise as a cure, the cell-type-specific heterogeneity of\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    expression in the lung presents significant challenges. Current CF ferret models closely replicate the human disease phenotype but have limitations in studying functional complementation through cell-type-specific CFTR restoration. To address this, we developed a new transgenic ferret line,\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    <jats:sup>int1-eGFP(lsl)<\/jats:sup>\n                    , in which a Cre-recombinase (Cre)-excisable enhanced fluorescent protein (eGFP) reporter cassette is knocked in (KI) to intron 1 of the\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    locus. Breeding this reporter line with\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    <jats:sup>G551D<\/jats:sup>\n                    CF ferret resulted in a novel CF model,\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    <jats:sup>int1-eGFP(lsl)\/G551D<\/jats:sup>\n                    , with disease onset manageable via the administration of CFTR modulator VX770. In this study, we confirmed two key properties of the\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    <jats:sup>int1-eGFP(lsl)\/G551D<\/jats:sup>\n                    CF ferrets: (1) cell-type-specific expression of the CFTR(N-24)-eGFP fusion protein, driven by the intrinsic\n                    <jats:italic toggle=\"yes\">CFTR<\/jats:italic>\n                    promoter, in polarized epithelial cultures and selected tissues, and (2) functional reversion of the KI allele via Cre-mediated excision of the reporter cassette. This model provides a valuable tool for studying the effects of targeted CFTR reactivation in a cell-type-specific manner, which is crucial for enhancing our understanding of CFTR\u2019s roles in modulating airway clearance and innate immunity, and for identifying relevant cellular targets for CF gene therapy.\n                  <\/jats:p>","DOI":"10.1089\/hum.2024.215","type":"journal-article","created":{"date-parts":[[2025,1,10]],"date-time":"2025-01-10T04:29:47Z","timestamp":1736483387000},"page":"1053-1066","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":6,"title":["Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR Reactivation"],"prefix":"10.1177","volume":"36","author":[{"given":"Feng","family":"Yuan","sequence":"first","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xingshen","family":"Sun","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soo Yeun","family":"Park","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yinghua","family":"Tang","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zehua","family":"Feng","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mehrnoosh","family":"Ebadi","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yaling","family":"Yi","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adriane E.","family":"Thompson","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph D.","family":"Karippaparambil","sequence":"additional","affiliation":[{"name":"Department of Anatomy and Cell Biology, Carver College of Medicine, University of Iowa, Iowa 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