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Am J Respir Cell Mol Biol. 2018 Dec;59(6):672-683. doi: 10.1165/rcmb.2018-0213TR.

Induced Pluripotent Stem Cells for Primary Ciliary Dyskinesia Modeling and Personalized Medicine.

Author information

1
1 Institute for Regenerative Medicine and Biotherapy, University of Montpellier, Institut National de la Santé et de la Recherche Médicale (INSERM), Centre Hospitalier Universitaire Montpellier, Montpellier, France.
2
2 PhyMedExp, University of Montpellier, INSERM, Centre Hospitalier Universitaire Montpellier, Montpellier, France; and.
3
3 Centre Hospitalier Universitaire Montpellier, Department of Cell and Tissue Engineering, Hospital Saint-Eloi, Montpellier, France.

Abstract

Primary ciliary dyskinesia (PCD) is a rare and heterogeneous genetic disorder that affects the structure and function of motile cilia. In the airway epithelium, impaired ciliary motion results in reduced or absent mucociliary clearance that leads to the appearance of chronic airway infection, sinusitis, and bronchiectasis. Currently, there is no effective treatment for PCD, and research is limited by the lack of convenient models to study this disease and investigate innovative therapies. Furthermore, the high heterogeneity of PCD genotypes is likely to hinder the development of a single therapy for all patients. The generation of patient-derived, induced pluripotent stem cells, and their differentiation into airway epithelium, as well as genome editing technologies, could represent major tools for in vitro PCD modeling and for developing personalized therapies. Here, we review PCD pathogenesis and then discuss how human induced pluripotent stem cells could be used to model this disease for the development of innovative, patient-specific biotherapies.

KEYWORDS:

PCD; airway epithelium; cilia; iPSC; therapy

PMID:
30230352
DOI:
10.1165/rcmb.2018-0213TR
[Indexed for MEDLINE]

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