Small molecule-facilitated anion transporters in cells for a novel therapeutic approach to cystic fibrosis.

ABSTRACT:

Background and purpose

Cystic fibrosis (CF) is a lethal autosomal recessive genetic disease that originates from the defective function of the CF transmembrane conductance regulator (CFTR) protein, a cAMP-dependent anion channel involved in fluid transport across epithelium. Because small synthetic transmembrane anion transporters (anionophores) can replace the biological anion transport mechanisms, independent of genetic mutations in the CFTR, such anionophores are candidates as new potential treatments for CF.

Experimental approach

In order to assess their effects on cell physiology, we have analysed the transport properties of five anionophore compounds, three prodigiosines and two tambjamines. Chloride efflux was measured in large uni-lamellar vesicles and in HEK293 cell

SUBMITTER: Fiore M 

PROVIDER: S-EPMC6514296 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature