Targeting the Lung: Challenges in Gene Therapy for Cystic Fibrosis

Cystic Fibrosis (CF) is the most common fatal autosomal recessive genetic disease in the Caucasians with a frequency of approximately 1 in 2500 newborns (Cystic Fibrosis Founda‐ tion, http://www.cff.org/). It affects several organs including the lungs, the liver, the pan‐ creas, the sweat glands and the gastrointestinal and reproductive tracts [1]. The most severe complications that finally lead to death are those in the airway epithelium [2]. Continuous secretion of mucus causes blockage of the lungs by thick sputum and also makes the lungs susceptible to secondary bacterial infections. Subsequent inflammatory responses by the im‐ mune system damage the lungs and the combination of all these factors leads to cardiac fail‐ ure and to death [3].