Molecular pharmacology of cystic fibrosis

Cystic fibrosis is caused by mutations in the CFTR protein which is an anion transporter of the apical membrane of epithelia. CFTR is an anion transporter of the apical membrane of epithelia. Mutations in the CFTR gene cause various defects, such as the inability to synthesize the protein, folding errors, defects in "gating" or conductance or reduced expression, leading to cystic fibrosis. We study the properties of drugs that improve the functioning of the transport (enhancers), or that recover the CFTR with refolding errors (correctors). We also study synthetic compounds that carry anions through the membranes, as replacement therapy in cystic fibrosis.

Members:
Debora Baroni
Michele Fiore