The genetics of cystic fibrosis: a paradigm for uncovering new drug targets.

A broad-based approach will be required for the development of new therapies for cystic fibrosis lung disease. Recently, rapid progress has been made in identifying and testing a number of gene transfer vectors, including adenoviral vectors and liposomes. Major problems, however, have been identified with respect to the efficiency of these systems. Preliminary studies suggest that small molecules (e.g. amelioride and UTP) may normalize the clearance of secretions from the cystic fibrosis lung. The concept of recombinant protein based therapy for cystic fibrosis has now been realized with the successful application of DNase in clinical trials.