Cystic fibrosis: recent advances in lung-directed therapies

Cystic fibrosis (CF) lung disease is characterized by progressively worsening pulmonary infections, airway obstruction and ultimately early death. Current pharmacotherapeutic treatments can only alleviate the secondary symptoms associated with lung disease. Alternative therapeutic strategies are now being developed to target the CF gene defect at the molecular level. Preclinical and clinical trials are currently underway and early results strongly suggest that these strategies can be translated into an effective curative therapy for CF lung disease.

Section editors:Frederick Kaplan – The University of Pennsylvania School of Medicine, Department of Orthopaedic Surgery, Philadelphia, PA 19104, USAAmber Salzman – Cardiokine, Inc., Philadelphia, PA 19102, USA