Cystic Fibrosis

April 2017

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. A group of drugs called modulators control the function of the CFTR protein so it can create a channel for chloride to flow across the cell surface. Amplifiers, a type of CFTR modulator, increase the amount of CFTR protein that the cell makes.


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