Pharmacological Responses of the G542X-CFTR to CFTR Modulators

Abstract excerpt

Cystic fibrosis (CF) is a lethal hereditary disease caused by loss-of-function mutations of the chloride channel cystic fibrosis transmembrane conductance regulator (CFTR). With the development of small-molecule CFTR modulators, including correctors that facilitate protein folding and expression and potentiators that promote channel activity, about 90% of the CF patients are now receiving efficacious target...

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