Teresinha Leal

Emerging Roles of microRNAs in Cystic Fibrosis — From Pathogenesis to Development of New Therapies

As essential components of the regulatory system of gene expression, microRNAs (miRNAs) have been shown to influence development, severity, prognosis, and/or progression of a variety of inherited diseases. Differential expression studies have evidenced an impact of miRNAs on lung disease development in chronic obstructive pulmonary disease (COPD), asthma, lung inflammation, consequences of smoke exposure and airway allergy in human and in animal models of the diseases. Recent clinical and cell-based studies have revealed specific alterations of miRNA expression in cystic fibrosis (CF). Here we critically review the major findings concerning altered miRNA expression in CF airway epithelium, in particular with respect to CF transmembrane conductance regulator (CFTR) expression, innate immunity, and epithelial differentiation. Finally, we explore strategies to exploit these changes with the aim of innovative therapeutic benefits.