Researchers in the University of Iowa Pappajohn Biomedical Institute have received a five-year, $11 million grant from the National Heart, Lung, and Blood Institute, part of the National Institutes of Health, to continue advancing gene therapy to treat and prevent cystic fibrosis, a life-shortening, lethal lung disease.

Cystic fibrosis is an inherited disease caused by a faulty gene (known as the CFTR gene). The CFTR gene normally creates a protein that moves salt and water out of a cell. If the CFTR gene is defective, it results in a build-up of thick, sticky mucus in the body. This leads to numerous problems, including lung infection and inflammation, the major causes of disease and death in cystic fibrosis.

Led by Paul McCray, M.D., professor of pediatrics, the UI team will use novel viruses to replace the defective CFTR gene in the airway cells and will develop new animal models to address questions regarding the specific cell types that must be corrected to prevent disease.

In addition to McCray, the team includes, David Stoltz, M.D., Ph.D., associate professor of internal medicine; Joseph Zabner, M.D., professor of internal medicine; Michael Welsh, M.D., professor of internal medicine, Howard Hughes Medical Institute Investigator, and director of the Pappajohn Biomedical Institute; Patrick Sinn, Ph.D., research associate professor of pediatrics and director of the UI Viral Vector Core; John Engelhardt, Ph.D., professor and chair of anatomy and cell biology; David Meyerholz, D.V.M., Ph.D., associate professor of pathology; and Lynda Ostedgaard, Ph.D., research associate professor of internal medicine.

The grant, “Gene Therapy for Cystic Fibrosis Lung Disease,” (Grant number 2P01HL051670) has been continuously funded since 1993.