What are the therapeutic strategies targeting CFTR in Cystic Fibrosis?
Several therapeutic strategies aim to correct or mitigate the effects of defective CFTR in CF patients. These include:
- CFTR modulators: Small molecules that improve the function of defective CFTR protein. - Gene therapy: Techniques designed to deliver a functional CFTR gene to affected cells. - Symptomatic treatments: Approaches like airway clearance techniques and enzyme replacement therapy to manage symptoms and complications.
