RUSNATI M.

Cystic fibrosis transmembrane conductance regulator (CFTR)-rescuing drugs have already transformed cystic fibrosis (CF) from a fatal disease to a treatable chronic condition. However, new-generation drugs able to bind CFTR with higher specificity/anity and to exert stronger therapeutic benefits and fewer side eects are still awaited....

Cystic Fibrosis (CF) is caused by mutations (mainly F508del) of the cystic fibrosis transmembrane conductance regulator (CFTR). Current CF therapies are aimed at symptoms alleviation, calling for new drugs to rescue CFTR function. Hypothesis and objectives Drug repositioning is aimed at finding new applications for already marketed drugs,...

Cystic fibrosis is a hereditary disease mainly caused by the deletion of the Phe 508 (F508del) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein that is thus withheld in the endoplasmic reticulum and rapidly degraded by the ubiquitin/proteasome system. Cystic fibrosis remains a potentially fatal disease, but it has...

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Cystic fibrosis (CF) is the autosomal recessive disorder most recurrent in Caucasian populations. It is caused by different mutations in the cystic fibrosis transmembrane regulator protein (CFTR) gene, with F508del being the most common. During the last years, small-molecule therapy chosen to contrast CF relied on compounds that correct CFTR...

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