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Peer-reviewedCC BY 4.0Cells

Conditionally Reprogrammed Patient-Derived Nasal Epithelial Cell Cultures for In Vitro Diagnostics and Pharmacological Profiling in Primary Ciliary Dyskinesia

  • Ruth M. Urbantat1,2,3,4
  • Anita Balázs1,2,4
  • Kathrin Seidel1,2,4
  • Marika Drescher1,2,4
  • Ricarda Lange1,2,4
  • Aditi Löwe1,2,4
  • Tihomir Rubil1,2,4
  • Julia Duerr1,2,4
  • Jobst F. Röhmel1,2,5
  • Stephanie Thee1,2,3,4
  • Mirjam Stahl1,2,3,4
  • Marcus A. Mall1,2,3,4,6
6 affiliations
  1. 1Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité—Universitätsmedizin Berlin, 13353 Berlin, Germany
  2. 2German Center for Lung Research (DZL), Associated Partner Site, 13353 Berlin, Germany
  3. 3Berlin Institute of Health (BIH), 10117 Berlin, Germany
  4. 4German Center for Child and Adolescent Health (DZKJ), Partner Site Berlin, 13353 Berlin, Germany
  5. 5Department of Respiratory and Critical Care Medicine, Charité—Universitätsmedizin Berlin, 10117 Berlin, Germany
  6. 6Cluster of Excellence ImmunoPreCept, Charité—Universitätsmedizin Berlin, 13353 Berlin, Germany
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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.

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Cite

BibTeXRIS
BibTeX
@article{urbantat2026conditionally,
  title = {{Conditionally Reprogrammed Patient-Derived Nasal Epithelial Cell Cultures for In Vitro Diagnostics and Pharmacological Profiling in Primary Ciliary Dyskinesia}},
  author = {Ruth M. Urbantat and Anita Balázs and Kathrin Seidel and Marika Drescher and Ricarda Lange and Aditi Löwe and Tihomir Rubil and Julia Duerr and Jobst F. Röhmel and Stephanie Thee and Mirjam Stahl and Marcus A. Mall},
  journal = {Cells},
  year = {2026},
  month = oct,
  doi = {10.3390/cells15201871},
  url = {https://www.mdpi.com/2073-4409/15/20/1871},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Conditionally Reprogrammed Patient-Derived Nasal Epithelial Cell Cultures for In Vitro Diagnostics and Pharmacological Profiling in Primary Ciliary Dyskinesia
AU  - Ruth M. Urbantat
AU  - Anita Balázs
AU  - Kathrin Seidel
AU  - Marika Drescher
AU  - Ricarda Lange
AU  - Aditi Löwe
AU  - Tihomir Rubil
AU  - Julia Duerr
AU  - Jobst F. Röhmel
AU  - Stephanie Thee
AU  - Mirjam Stahl
AU  - Marcus A. Mall
PY  - 2026
DA  - 2026/10/15
JO  - Cells
DO  - 10.3390/cells15201871
UR  - https://www.mdpi.com/2073-4409/15/20/1871
AB  - 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.
SN  - 2073-4409
ER  -