Article
CFTR Modulators: From Mechanism to Targeted Therapeutics.
Handbook of experimental pharmacology - 1 Jan 2024
Yeh Han-I, Sutcliffe Katy J, Sheppard David N, Hwang Tzyh-Chang
Abstract excerpt
People with cystic fibrosis (CF) suffer from a multi-organ disorder caused by loss-of-function variants in the gene encoding the epithelial anion channel cystic fibrosis transmembrane conductance regulator (CFTR). Tremendous progress has been made in both basic and clinical sciences over the past three decades since the identification of the CFTR gene. Over 90% of people with CF now have access to therapies...
Topics
- Humans
- Cystic Fibrosis Transmembrane Conductance Regulator
- Mutation
- Cystic Fibrosis
- Signal Transduction
