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.