Lung disease in cystic fibrosis
| Problems | Solutions | Treatments |
|---|---|---|
| Mutation of CFTR | Provide normal gene | Gene therapy |
| Overcome stop mutations | Aminoglycosides | |
| Abnormal processing of CFTR | Relocalisation | Molecular chaperones |
| Defective CFTR | Upregulate function | Pharmacological agents |
| Abnormal ion transport | Increase chloride transport | UTP/ATP |
| Block sodium uptake | Amiloride | |
| Thick obstructive mucus | Decrease viscosity | Mucolytics |
| DNAse | ||
| Impaired clearance | Augment clearance | Chest physiotherapy |
| Pulmonary infection | Decrease bacterial load | Antibiotics |
| Salt-insensitive antimicrobial peptides | ||
| Pulmonary inflammation | Diminish host reaction | Antiprotease |
| Bronchiectasis | Replace when irreversible damage | Lung transplant |