Article
Targeting CFTR: how to treat cystic fibrosis by CFTR-repairing therapies.
Current drug targets - 1 May 2011
Amaral Margarida D
Abstract excerpt
Several novel compounds recently appeared as promising leads to develop effective drugs against the basic defect in Cystic fibrosis (CF) and the first rationale therapies for CF relying on the understanding of the basic defect started to hit the clinical setting. Most of these efforts are focused on correcting the F508del mutation (occurring in ≈90% of CF patients) which causes misfolding of the CF transmembrane...
Topics
- Animals
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Drug Discovery
- Female
- Genetic Therapy
- Humans
- Male
- Mice
- Molecular Targeted Therapy
- Mutation
- Protein Folding