Testing CFTR repair in cystic fibrosis patients carrying nonsense and channel gating mutations

Abstract

Background. In order to measure CFTR activity in a minimal invasive manner, we set up a method to assay functional CFTR and focussed on leukocytes from healthy and CF donors. Leukocytes are recognized in the scientific literature as a key component of the pathogenetic events associated to cystic fibrosis and represent an easily accessible source of primary cells that might be exploited to monitor CFTR expression and activity.