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

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 (iPSC) and their differentiation into airway epithelium as well as genome editing technologies could represent major tools for in vitro PCD modelling and for developing personalized therapies. Here, we review PCD pathogenesis, and then discuss how human iPSC could be used to model this disease for the development of innovative patient-specific biotherapies.
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Soumis le : lundi 20 janvier 2020 - 12:51:20
Dernière modification le : mercredi 22 janvier 2020 - 11:37:29

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Joffrey Mianné, Engi Ahmed, Chloé Bourguignon, Mathieu Fieldes, Isabelle Vachier, et al.. Induced Pluripotent Stem Cells for Primary Ciliary Dyskinesia Modeling and Personalized Medicine. American Journal of Respiratory Cell and Molecular Biology, American Thoracic Society, 2018, 59 (6), pp.672-683. ⟨10.1165/rcmb.2018-0213TR⟩. ⟨hal-01878063⟩

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