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    ISSN : 2328-9791
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Alternative strategies for F508del-CFTR repair: novel targets for drug discovery approach in Cystic Fibrosis
     
 
Author(s) : Cozza G, Tosco A, Ferrari E, Villella VR, Esposito S, Monzani R, Spinella MC, Venerando A, Rossetto M, Bosello Travain V, Ursini F, Raia V, Maiuri L
Address : Multiple universities in Milan, Padua and Naples, Italy.
flaminia.malvezzi@fibrosicisticaricerca.it

Background and Rationale. Cystic Fibrosis (CF) is a life-shortening genetic disorder, caused by mutations of Cystic Fibrosis Transmembrane-conductance Regulator (CFTR) [1]. In particular, the most common F508delCFTR mutant is unable to traffic to and reside at the plasma membrane (PM) [2]. Currently, no highly effective CFTR-repairing therapies are available for F508del-CFTR. For this reason our novel approach aims at targeting the derailed CF intracellular environment, and not directly the mutant CFTR, through a combination of drugs (e.g. cysteamine and epigallocatechin-gallate, EGCG) able to inhibit TG2/PDI activities and specific protein kinases [3].   

Hypothesis and Objectives. We aim to 1) Refine new targets as novel therapeutic strategy in CF by exploiting a network of in silico and experimental approaches; 2) Validate the efficacy of novel drug candidates in pre-clinical CF models.  

 
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