This project, an extension of FFC#4/2019, aims to develop new molecules or their combinations capable of restoring the function of CFTR. Thanks to the results of previous projects, the researchers observed that oxidative stress is closely linked to defects in autophagy in cystic fibrosis (CF). Researchers identified a number of molecules capable of restoring CFTR function by balancing the pro-oxidative state in CF models. Thanks also to computational approaches such as Virtual Screening and Pharmacophoric search, the researchers will select molecules and validate their efficacy in both cellular experimental models, such as primary nasal epithelial cells from F508del, N1303K, G85E and R1066C donors and in vivo with transgenic mouse models.

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