Studies on Airway Inflammation and Remodeling in a Cystic Fibrosis F508del CFTR Mouse Model: Towards the Development of Experimental Therapies for the Treatment of the CF Lung Pathophysiology

Abstract

Cystic Fibrosis (CF) is the most common autosomal recessive life-threatening disease with short life expectancy, high incidence and prevalence worldwide. CF is caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene which mainly affects the fluid transport of secretory epithelial cells of several organs including the lungs. Chronic inflammation and associated irreversible airway remodeling, which eventually results in fatal loss of lung function, are the major cause of the high morbidity and mortality seen in CF. The complex CF lung pathophysiology is still poorly understood and an effective cure is still not available. The primary aim of the work presented in this thesis is to determine which molecular pathways are involved in the CF lung pathophysiology in order to use these in the future as potential targets for the development of novel diagnostic or therapeutic strategies for CF, and possibly other lung diseases.