Cystic fibrosis (CF) is a progressive genetic condition and is the result of a patient inheriting a faulty cystic fibroses transmembrane conductance regulator (CFTR) gene from both parents. The defective CFTR genes produce faulty CFTR proteins on the body’s epithelial cells, and result in the buildup of a thick and sticky mucus in the lungs and pancreas. Historically therapeutic options have focused heavily on treating the symptoms. 更多
