Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment

The basic defect in cystic fibrosis (CF) reflects loss of function of the cystic fibrosis transmembrane conductance regulator (CFTR), a plasma membrane protein involved in chloride and bicarbonate transport across cellular membranes. A key therapeutic strategy for CF focuses on CFTR modulators, drugs designed to improve the function of abnormal CFTR. All currently approved CFTR modulators include the potentiator compound ivacaftor (discovered as VX-770, [1]), which improves the gating efficiency of CFTR.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research