The treatment with Kaftrio and other Cystic Fibrosis (CF) drugs appears to modify the lipid and protein content of cells; on this observation, the researchers intend to study the alterations in proteins and lipids of the bronchial epithelium from primary cells obtained from persons with CF with the F508del mutation. These perturbations in lipid and protein content will be evaluated in association with treatment with Kaftrio. The researchers will study the bronchial epithelium of 10 persons with CF with the F508del mutation and 4 with “minimal function” mutations and compare them with those obtained from 10 non-CF control subjects. Advanced bioinformatics tools will be used to study the network of lipids and proteins specifically associated with CFTR recovery. The goal of this project is to deepen the knowledge of the mechanisms underlying the recovery of CFTR, useful for the design of new drugs for CF.

XIX Convention FFC Ricerca – download here a brief presentation of the project