Genetics On the Forefront of New Cystic Fibrosis Treatment

Another treatment method that could prove to be successful in treating CF from a genetic standpoint is the development of genotype-specific small molecule drugs that could change CFTR function. One category of these drugs is called potentiators (such as ivacaftor). Potentiators increase the function of the CFTR channels and would thus decrease the symptoms related to CFTR mutations. The second class of these drugs is called correctors (such as the drug lumacaftor). Correctors specifically target a type of CFTR mutation improving intracellular processing and allowing more of the protein the CF patients need from this gene, to reach the surface of the cell. The third type of molecular drugs are call production correctors or read-through agents. These drugs would work by allowing more CFTR protein to reach the cell surface in yet another type of CFTR mutation.