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C2TN

A MOLECULAR IMAGING APPROACH TO CYSTIC FIBROSIS

G1 8

 

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR), which encodes a chloride channel present at the plasma membrane of epithelial cells. Although being one of the most intensively investigated monogenic disorders, symptomatic treatment remained the bedrock of CF patient care until the beginning of this decade. Recently, novel CF therapies based on small organic molecules that target the basic defect in CF were approved by the EMA/FDA. Despite this exciting development, from the translational medicine perspective, there remains a need to improve disease biomarkers and non-invasive therapeutic endpoints used in clinical trials. Within our goal of bringing forward new imaging biomarkers for CF management, we have explored a small-molecule based approach for the development of non-invasive probes. Currently, we are expanding these studies to other CFTR-targeting biomolecules, and in collaboration with iMed.Ulisboa, Fabs will be selected from a human library by phage display.