Cystic fibrosis

Abstract

Cystic fibrosis is a genetic disease that affects multiple organ systems, yet leads to respiratory failure and premature death in most afflicted patients. Rapid advancements in our understanding of disease pathogenesis have occurred because of the cloning of the cystic fibrosis transmembrane conductance regulator gene. Recent work has shown that volume depletion of the periciliary fluid layer and mucus dehydration reduces mucus clearance and promotes the initiation and progression of CF lung disease. Therapies that correct dysregulated salt and water transport in the lung, either by correcting or bypassing the basic cystic fibrosis trans-membrane conductance regulator defect, are now being developed and hold great promise. © 2006 Humana Press Inc.