Model systems to Assess The in vitro efficacy of CFTR modulator tHerapies in Cystic Fibrosis (MATCH-CF)
Cystic fibrosis (CF) is a genetic disease is caused by changes (mutations) in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). CFTR is responsible for salt transport and when it does not work properly it leads to organ dysfunction. A new generation of therapies are now available that improve function of the CFTR protein and are known as CFTR modulators and they have contributed to improving the lung function and the quality of life for people living with CF (PwCF). However, a minority of PwCF may benefit from these drugs but do not currently have access due to their rare genotype. Our goal is to develop tests to predict individual clinical responses to these CFTR modulators. The expected benefits to the partner organizations are to develop renewable and long-living human airway cells from PwCF so CF researchers can evaluate the response to CFTR modulating drugs and develop high-throughput drug discovery screens.