Article
Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis.
Seminars in respiratory and critical care medicine - 1 Dec 2019
Cuyx Senne, De Boeck Kris
Abstract excerpt
Detailed knowledge of how mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene disturb the trafficking or function of the CFTR protein and the use of high-throughput drug screens have allowed novel therapeutic strategies for cystic fibrosis (CF). The main goal of treatment is slowly but surely shifting from symptomatic management to targeting the underlying CFTR defect to halt disease...
Topics
- Aminophenols
- Aminopyridines
- Benzodioxoles
- Clinical Trials, Phase II as Topic
- Clinical Trials, Phase III as Topic
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Genetic Therapy
- Humans
- Indoles