CFTR inhibitors

CFTR inhibitors are pharmacological agents that target the cystic fibrosis transmembrane conductance regulator (CFTR), a protein involved in chloride ion transport across cell membranes. CFTR is essential for maintaining fluid balance in various epithelial tissues, including the lungs, pancreas, and intestines. Mutations in the CFTR gene lead to defective or dysfunctional CFTR protein, resulting in cystic fibrosis (CF), a genetic disorder characterized by thick, sticky mucus production and impaired organ function. CFTR inhibitors work by blocking or reducing CFTR activity, which can help modulate ion transport and potentially alleviate symptoms associated with CF.