CFTR modulators work by targeting the defective CFTR protein. There are different types of modulators, including potentiators, correctors, and amplifiers:
Potentiators: These enhance the function of the CFTR protein that reaches the cell surface. They help to increase the gating activity of the protein, allowing more chloride ions to pass through. Correctors: They assist in the proper folding and trafficking of the CFTR protein to the cell surface. Correctors address the misfolding that often results from certain CFTR mutations. Amplifiers: These increase the amount of CFTR protein produced by the cell, thereby enhancing its overall function.
