{
  "abstract": "Background: Air-liquid interface (ALI) cultures of patient-derived primary nasal epithelial cells (pNEC) can support diagnostics and in vitro phenotyping of primary ciliary dyskinesia (PCD); however, low cell yield remains a limitation. We aimed to establish and optimize conditionally reprogrammed cell (CRC) technology for pNEC from individuals with PCD and use it as a diagnostic tool for inconclusive cases and for pharmacological testing. Methods: pNEC from 36 individuals (8 healthy controls (HC), 26 PCD, 2 PCD candidates) were expanded using CRC and differentiated at ALI. Ciliary beat frequency (CBF), mucociliary transport velocity (MCTV), and culture morphology were assessed. Further, we assessed the effect of the β-receptor agonist clenbuterol on cilia function in pNEC of HC, PCD (DNAH11, CCDC40) and PCD candidates harboring biallelic DNAH1 variants. Results: CRC expansion demonstrated a >90% success rate, yielding high cell counts (mean 9.65 × 106). Differentiation at ALI resulted in pseudostratified ciliated pNEC cultures. CBF was comparable between native and cultured pNEC. MCTV was significantly reduced in PCD vs. HC cultures (p < 0.0001). Clenbuterol significantly increased CBF in HC and DNAH1, but not in CCDC40 or DNAH11 cultures. MCTV was significantly increased in HC by clenbuterol treatment. Conclusions: CRC technology provides a scalable platform for the expansion of pNEC that recapitulate in vivo ciliary function. By enabling genotype-specific pharmacological screening, CRC may serve as a tool for functional diagnostics and precision medicine in PCD.",
  "abstract_shown": true,
  "affiliations": [
    "Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité—Universitätsmedizin Berlin, 13353 Berlin, Germany",
    "German Center for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany",
    "Berlin Institute of Health (BIH), 10117 Berlin, Germany",
    "German Center for Child and Adolescent Health (DZKJ), Partner Site Berlin, 13353 Berlin, Germany",
    "Department of Respiratory and Critical Care Medicine, Charité—Universitätsmedizin Berlin, 10117 Berlin, Germany",
    "Cluster of Excellence ImmunoPreCept, Charité—Universitätsmedizin Berlin, 13353 Berlin, Germany"
  ],
  "article_type": "research article",
  "arxiv": null,
  "author_total": 12,
  "authors": [
    {
      "affs": [
        0,
        1,
        2,
        3
      ],
      "name": "Ruth M. Urbantat",
      "orcid": "0000-0001-7360-6001"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Anita Balázs",
      "orcid": "0000-0002-1402-5239"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Kathrin Seidel"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Marika Drescher"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Ricarda Lange"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Aditi Löwe",
      "orcid": "0009-0002-6561-6589"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Tihomir Rubil",
      "orcid": "0000-0003-3346-4031"
    },
    {
      "affs": [
        0,
        1,
        3
      ],
      "name": "Julia Duerr",
      "orcid": "0000-0002-4862-9487"
    },
    {
      "affs": [
        0,
        1,
        4
      ],
      "name": "Jobst F. Röhmel"
    },
    {
      "affs": [
        0,
        1,
        2,
        3
      ],
      "name": "Stephanie Thee",
      "orcid": "0000-0001-9090-7869"
    },
    {
      "affs": [
        0,
        1,
        2,
        3
      ],
      "name": "Mirjam Stahl",
      "orcid": "0000-0001-6941-8315"
    },
    {
      "affs": [
        0,
        1,
        2,
        3,
        5
      ],
      "name": "Marcus A. Mall",
      "orcid": "0000-0002-4057-2199"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/0cjdh2xaxqm14.bib",
  "cited_by": 0,
  "doi": "10.3390/cells15201871",
  "fulltext_url": "https://europepmc.org/articles/PMC13703304",
  "id": "0cjdh2xaxqm14",
  "issns": [
    "2073-4409"
  ],
  "landing_url": "https://www.mdpi.com/2073-4409/15/20/1871",
  "language": "eng",
  "licence": "CC-BY-4.0",
  "licence_label": "CC BY 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "openalex",
      "2740|Cystic Fibrosis Research Advances"
    ]
  ],
  "openalex": "W7222182309",
  "pdf_url": "https://europepmc.org/articles/PMC13703304?pdf=render",
  "pmcid": "PMC13703304",
  "pmid": "42929845",
  "published": "2026-10-15",
  "publisher": "MDPI AG",
  "ris": "https://research.draconitic.com/paper/0cjdh2xaxqm14.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:19:02Z",
      "native_id": "10.3390/cells15201871",
      "source": "crossref",
      "url": "https://www.mdpi.com/2073-4409/15/20/1871"
    },
    {
      "first_seen": "2026-10-30T12:22:01Z",
      "native_id": "MED:42929845",
      "source": "europepmc",
      "url": "https://europepmc.org/article/MED/42929845"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "issn:2073-4409",
      "subfield": "cell-biology"
    }
  ],
  "title": "Conditionally Reprogrammed Patient-Derived Nasal Epithelial Cell Cultures for In Vitro Diagnostics and Pharmacological Profiling in Primary Ciliary Dyskinesia",
  "title_text": "Conditionally Reprogrammed Patient-Derived Nasal Epithelial Cell Cultures for In Vitro Diagnostics and Pharmacological Profiling in Primary Ciliary Dyskinesia",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:22:01Z",
  "url": "https://research.draconitic.com/paper/0cjdh2xaxqm14",
  "venue": "Cells",
  "versions": []
}