John F. Engelhardt, Ph.D., is highly regarded for his research focused on the molecular basis of cystic fibrosis (CF) disease pathologies and on the development of gene therapies for this disorder.
His work spans four major research areas:
- lung molecular and cellular biology as it relates to the pathogenesis and treatment of CF
- the development of viral vector for gene therapy and gene editing
- pathogenesis cystic fibrosis related diabetes
- the study of airway stem cell niches, the regulatory mechanisms that control stem cell proliferation and repair in the airway, and the development of cell-based therapies for CF using stem cells
Over the course of three decades in academic medicine, Dr. Engelhardt has defined how the cystic fibrosis transmembrane conductance regulator (CFTR) channel coordinates fluid homeostasis at the airway surface through specialized rare cells called ionocytes and airway submucosal glands. His work has also elucidated Wnt-mediated mechanisms that facilitate airway repair by submucosal gland myoepithelial stem cells. Unlike mice, humans and ferrets contain submucosal glands throughout the cartilaginous airways, and thus he pioneered the creation of genetically engineered ferrets to study CF pathogenesis and stem cell biology in the lung and pancreas – two organs that lack pathology in CF mice.
This research has enabled Cre/Lox technologies in ferrets, which he has used to conditionally delete CFTR in ionocytes and fate map airway gland and pancreatic stem cells in the setting of CF disease. He directs a federally funded National Ferret Resource Center serving the greater scientific community and biotech sector across a range of lung and pancreatic diseases.
Dr. Engelhardt’s work on CF-related diabetes in humans and ferrets has uncovered new biology governing early pancreatic remodeling, islet death and regeneration, and molecular processes that control these events. His virology and gene therapy research has defined molecular pathways that regulate adeno-associated virus trafficking and genome persistence.
As of December 2024, he had a lifetime $106 million total of NIH support as Principal Investigator. Dr. Engelhardt has published 304 manuscripts in peer reviewed journals, and 31 manuscripts in non-peer reviewed journals, reviews, and book chapters. As reported in Google Scholar, Dr. Engelhardt currently has an H-index of 110 with over 39,000 citations.
Dr. Engelhardt received his B.S. from Iowa State University and Ph.D. from Johns Hopkins University. Upon earning his Ph.D., he completed his postdoctoral work at the University of Michigan before joining the faculty at the University of Pennsylvania. He has been a faculty member of the Department of Anatomy and Cell Biology at the University of Iowa since 1997 and served as Department Chair from 2004-2024. He has been the Director of the University of Iowa Center for Gene Therapy for the past 25 years and holds the Endowed Carver Chair in Molecular Medicine.
He joins the UAB Division of Pulmonary, Allergy and Critical Care Medicine as Professor of Medicine.