title: New insights about miRNAs in cystic fibrosis.
abstract: The molecular basis of cystic fibrosis (CF) is a mutation-related defect in the epithelial-cell chloride channel called CF transmembrane conductance regulator (CFTR). This defect alters chloride ion transport and impairs water transport across the cell membrane. Marked clinical heterogeneity occurs even among patients carrying the same mutation in the CFTR gene. Recent studies suggest that such heterogeneity could be related to epigenetic factors and/or miRNAs, which are small noncoding RNAs that modulate the expression of various proteins via post-transcriptional inhibition of gene expression. In the respiratory system, it has been shown that the dysregulation of miRNAs could participate in and lead to pathogenicity in several diseases. In CF airways, recent studies have proposed that miRNAs may modulate disease progression by affecting the production of either CFTR or various proteins that are dysregulated in the CF lung. Herein, we provide an overview of studies showing how miRNAs may modulate CF pathology and the efforts to develop miRNA-based treatments and/or to consider miRNAs as biomarkers. The identification of miRNAs involved in CF disease progression opens up new avenues toward treatments targeting selected clinical components of CF, independently from the CFTR mutation.
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France.
authors: Florence
authors: F
authors: Sonneville
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France.
authors: Manon
authors: M
authors: Ruffin
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France.
authors: Loïc
authors: L
authors: Guillot
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France.
authors: Nathalie
authors: N
authors: Rousselet
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France.
authors: Philippe
authors: P
authors: Le Rouzic
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France; Paediatric Respiratory Department, Hôpital Trousseau, AP-HP, Paris, France.
authors: Harriet
authors: H
authors: Corvol
authors: Inserm UMR_S938, CDR Saint-Antoine, Paris, France; Sorbonne Universités, UPMC University Paris 06, Paris, France. Electronic address: olivier.tabary@inserm.fr.
authors: Olivier
authors: O
authors: Tabary
mesh_terms: D000818:Animals; D015415:Biomarkers / Q000378:metabolism; D019295:Computational Biology; D003550:Cystic Fibrosis / Q000235:genetics* / Q000503:physiopathology / Q000628:therapy; D005786:Gene Expression Regulation; D020022:Genetic Predisposition to Disease; D006801:Humans; D035683:MicroRNAs / Q000235:genetics* / Q000378:metabolism
chemical_list: D015415:Biomarkers; D035683:MicroRNAs