Most of the problems associated with the disease cystic fibrosis depend on a non-correct folding of the CFTR in the ER and a consequent low level of expression at the cell surface. Our research efforts are therefore aimed at exploring a prototype therapeutic strategy to promote and enhance deltaF508-CFTR protein folding and its expression at the plasma membrane. To this scope disarmed toxins will be targeted to the ER alone or linked with chemical chaperones. By this strategy we will exploit existing knowledge on the ability of “chemical chaperones” that were found to correct Class II trafficking defects in deltaF508-CFTR. By directing and concentrating them to the ER by linking to disarmed toxins we intend to improve their selectivity of action, to decrease their general toxicity and to propose a novel therapeutic intervention in the cystic fibrosis. Prof. L.M. Roberts (Department of Biological Sciences, University of Warwick, Coventry-UK) also partecipates to this project.
