CFTR modulator
CFTR modulators are a class of drugs designed to treat cystic fibrosis (CF) by targeting the underlying genetic defect in the CFTR gene. Cystic fibrosis transmembrane conductance regulator (CFTR) modulators work by correcting the function of the defective CFTR protein, which is responsible for regulating chloride ion transport across cell membranes in various tissues such as the lungs, pancreas, and intestines. CFTR modulators can enhance CFTR protein activity in individuals with specific mutations of the CFTR gene, thereby improving chloride ion transport and reducing the production of thick mucus that characterizes CF.
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