Repairing the basic defect in cystic fibrosis - one approach is not enough.

Abstract excerpt

Cystic fibrosis has attracted much attention in recent years due to significant advances in the pharmacological targeting of the basic defect underlying this recessive disorder: the deficient functional expression of mutant cystic fibrosis transmembrane conductance regulator (CFTR) chloride channels at the apical membrane of epithelial cells. However, increasing evidence points to the reduced efficacy of single...

Topics

• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Genetic Therapy
• Humans
• Molecular Targeted Therapy
• Mutation
• Protein Folding
• Protein Processing, Post-Translational
• Protein Transport