Precision therapy of cystic fibrosis (CF) using CFTR modulators is now a reality for some pathogenetic variants, but the efficacy on a large number of rare, often individual, variants is unknown. By “theratyping” we mean the experimentation, at cellular level, of drugs already in clinical use for CF, on genotypes not previously evaluated and for which they were not designed. The aim of this project is to evaluate therapies already in use on rare genotypes of CFTR using nasal airway epithelial stem cells from persons with CF and corresponding organoids (theratyping). At the cellular level, the possibility of increasing the amount of CFTR on which modulators can act (“amplifying therapy”) will also be evaluated and a new combination of modulators will be tested. The results of the project can be used as a starting point for future clinical studies.

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