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University of Iowa News Release

Release: Oct. 8, 2003

(Photo: John Engelhardt, Ph.D., UI professor of anatomy and cell biology and director of the University of Iowa Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases)

UI Gene Therapy Center Receives $5.3 Million Grant

The University of Iowa Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases has received a 5-year, $5.3 million grant renewal from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), one of the National Institutes of Health (NIH).

The center was established in 1998 through joint funding by NIDDK and the Cystic Fibrosis Foundation to promote research and training on gene therapy approaches for treating cystic fibrosis (CF) and other devastating inherited diseases. John Engelhardt, Ph.D., UI professor of anatomy and cell biology, is the center's director and Beverly Davidson, Ph.D., the Roy J. Carver Professor in Internal Medicine and professor of neurology, and physiology and biophysics, is associate director.

In the first five years of its existence, the center has sponsored 33 pilot projects for a total of $3 million. These pilots have supported the work of many young researchers and established or expanded specialized research facilities for gene therapy research used by UI researchers and scientists from collaborating institutions.

Engelhardt explained that the main emphasis of the center is to provide infrastructure for researchers in the area of gene therapy in cystic fibrosis. In particular, the center runs and finances several research cores that handle some of the specialized experimental procedures commonly used in gene therapy and molecular medicine research.

These cores include the vector core, which designs and makes recombinant viruses to deliver genes; the animal model core, which develops mostly mouse model systems to study disease processes and to test gene therapies; the morphology core that analyzes tissue and cells to determine the expression of genes and proteins in various model systems; and a core that collects and utilizes CF lung tissue donated by CF patients who have undergone lung transplants.

"The CF lung tissue donated by these patients is an extremely integral and useful component of our center. This tissue allows us to build models to study disease process in human CF cells and test the gene-based therapies we develop within the center," Engelhardt said.

Joseph Zabner, M.D., UI associate professor of internal medicine, and Michael Welsh, M.D., the Roy J. Carver Biomedical Research Chair in Internal Medicine and Physiology and Biophysics, UI professor, and Howard Hughes Medical Institute Investigator, are co-directors of this unique and specialized core, which collaborates with nine lung transplant centers to acquire the CF lung tissue for research.

Paul McCray, M.D., UI professor of pediatrics, and Kenneth Moore, Ph.D., director of the UI Central Microscopy Facility, co-direct the morphology core, Engelhardt and Curt Sigmund, Ph.D., UI professor of internal medicine and physiology and biophysics, co-direct the animal core, and Davidson directs the vector core.

In addition to the collaborations with the lung transplant centers, which have led to many new intra-institutional projects, researchers at the UI Center for Gene Therapy also collaborate with several private companies to advance gene therapy technologies. These collaborations point to the center's success in strengthening research programs in the area of molecular medicine and gene therapy.

Engelhardt also noted that in the in the past five years, the center's membership has grown from 48 to 73, indicating that the center is fulfilling another aspect of its mission to attract more researchers to the UI's gene therapy efforts.

In addition to research, another important component to the center is its education mission. Many graduate students and postdoctoral researchers as well as faculty members receive training in the area of molecular medicine and gene therapy. The center's web site (http://genetherapy.genetics.uiowa.edu/) also provides the public with access to information on various genetic diseases and on the latest gene therapy research.

University of Iowa Health Care describes the partnership between the UI Roy J. and Lucille A. Carver College of Medicine and UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide. Visit UI Health Care online at www.uihealthcare.com.

STORY SOURCE: University of Iowa Health Science Relations, 5141 Westlawn, Iowa City, Iowa 52242-1178

CONTACT: Jennifer Brown, (319) 335-9917 jennifer-l-brown@uiowa.edu

PHOTOS/GRAPHICS: A photo of John Engelhardt is available from Jennifer Brown at jennifer-l-brown@uiowa.edu