An update on new and emerging therapies for cystic fibrosis.

Abstract excerpt

INTRODUCTION: Cystic fibrosis (CF) is a genetic disorder that results in a multi-organ disease with progressive respiratory decline that ultimately leads to premature death. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for the CFTR anion channel. Established CF treatments target downstream manifestations of the primary genetic defect, including...

Topics

• Animals
• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Drug Design
• Humans
• Molecular Targeted Therapy
• Mutation