Article
A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect.
The Lancet. Respiratory medicine - 1 Apr 2013
Boyle Michael P, De Boeck Kris
Abstract excerpt
Cystic fibrosis is caused by dysfunction or deficiency of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, an epithelial chloride channel that has a key role in maintaining homoeostasis of the airway surface liquid layer in the lungs. More than 1900 CFTR mutations that might result in a disease phenotype have been identified; these can be grouped into classes on the basis of their effect on...
Topics
- Aminophenols
- Biological Transport
- Clinical Trials as Topic
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Forecasting
- Humans
- Mutation
- Oxadiazoles
- Precision Medicine
- Protein Biosynthesis
- Quinolones
- Respiratory System Agents
- Treatment Outcome