A synthetic chloride channel restores chloride conductance in human cystic fibrosis epithelial cells

Abstract

Mutations in the gene-encoding cystic fibrosis transmembrane conductance regulator (CFTR) cause defective transepithelial transport of chloride (Cl -) ions and fluid, thereby becoming responsible for the onset of cystic fibrosis (CF). One strategy to reduce the pathophysiology associated with CF is to increase Cl - transport through alternative pathways. In this paper, we demonstrate that a small synthetic molecule which forms Cl - channels to mediate Cl - transport across lipid bilayer membranes is capable of restoring Cl - permeability in human CF epithelial cells; as a result, it has the potential to become a lead compound for the treatment of human diseases associated with Cl - channel dysfunction. © 2012 Shen et al.