Gene therapy for cystic fibrosis lung disease

Gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF), suggests new research. Working with CF pigs, the researchers have shown that two different virus-based vectors can restore a working version of the cystic fibrosis transmembrane conductance regulator (CFTR) protein that is faulty in CF to the pigs’ airway cells. Moreover, this gene replacement normalized important aspects of the lung biology and improved the ability of airway secretions to kill bacteria.