Gene therapy shows promise for cystic fibrosis

University of Iowa researchers have shown that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF).

Working with a pig model of CF, teams led by Paul McCray Jr. (’81 MD, ’84 R, ’88 F), professor of pediatrics, and Joseph Zabner, MD, professor of internal medicine, showed that two different virus-based vectors can restore a working version of a protein—the cystic fibrosis transmembrane conductance regulator (CFTR)—that is faulty in CF to the pigs’ airway cells. Moreover, this gene replacement normalizes important aspects of the lung biology and improves the ability of airway secretions to kill bacteria.

McCray’s team used a lentivirus while Zabner’s team used an adeno-associated virus (AAV). Both viruses have proved safe for use in humans. However, lentiviruses have not been tested in human lungs, and AAVs do not produce permanent gene expression. The studies were published in JCI Insight.