Abstract: Cystic fibrosis transmembrane conductance regulator (CFTR) is a chloride (Cl−) channel regulated by phosphorylation and intracellular nucleotides. CFTR is the major Cl− transport pathway in airway epithelial cells. The abnormal transepithelial Cl− transport and subsequent defective fluid transport caused by CF is a result of the genetic mutations of the gene coding for the CFTR protein. The present invention is directed to the novel use of ionophores as artificial Cl− transport pathways into CF epithelia to treat the defective Cl− and fluid transport lonophores and in particular, small molecule ionophores, represent a potential novel means of treating CF. The invention also includes using an ionophore to generate chloride secretion on intact monolayers of airway epithelia cells and other epithelia cells by administering an ionophore to a mammal.

Abstract: Methods and compositions for treating CF by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.

Abstract: Methods and compositions for treating CF by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.