Development and Investigation of Dry Powder Inhalers for Cystic Fibrosis

Cystic Fibrosis (CF) is the most common lethal monogenic disorder in Caucasians, estimated to affect one per 2500-4000 newborns. CF is caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) [1, 2]. CFTR acts mainly as a chloride channel and has other regulatory roles, including inhibition of sodium transport through the epithelial sodium channel, regulation of ATP channels and intracellular vesicle transport, acidification of intracellular organelles and inhibition of endogenous calciumactivated chloride channels [3-5]. CFTR is also involved in bicarbonate-chloride exchange [6]. In the airways, loss of functional CFTR promotes increase of oxidation status, tissue injury, modification of intracellular signaling pathways, cell apoptosis and inflammatory processes.