LAUSR

Cystic fibrosis (CF), one of the most common lethal genetic diseases in those of European descent, is caused by loss-of-function mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene, which encodes an epithelial ion channel, CFTR, that mediates chloride and bicarbonate transport across the cell membrane [1]. Patients with CF suffer from multiorgan dysfunctions, but mortality and morbidity are mainly caused by progressive respiratory impairment. 30 years after the breakthrough discovery of the CFTR gene, we are now witnessing the success of precision medicine in CF clinics prescribing small-molecule compounds targeting the CFTR protein [2]. These CFTR modulators have brought transformative effects on the health and well-being of CF patients and their families. F508del-CFTR, the most common disease-associated mutation in cystic fibrosis, may lose its responsiveness to the CFTR potentiator ivacaftor upon prolonged exposure to the newly developed CF drug Trikafta https://bit.ly/3A22MKQ