Understanding CFTR catalysis has significant therapeutic implications. Drugs known as CFTR modulators have been developed to enhance the function of defective CFTR proteins. These include:
- Potentiators: These drugs, like ivacaftor, enhance the gating function of CFTR, increasing the probability that the channel remains open. - Correctors: These agents aid in the proper folding and trafficking of CFTR to the cell surface. An example is lumacaftor. - Amplifiers: These increase the amount of CFTR protein produced by the cell.
Combining these drugs can be particularly effective for treating patients with specific CFTR mutations.
