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University of Iowa News Release

July 26, 2006

UI Gene Therapy Center Awards Seed Grants

The University of Iowa Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases has awarded grants for six pilot projects aimed at advancing gene therapy research. Five UI scientists and one from the University of Toronto each will receive $32,000.

The pilot program has been running for eight years and is funded through a center grant from the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health. The pilot grants provide seed money for researchers to pursue innovative but untested ideas related to gene therapy. Results from such pilot studies can open up new areas of research and often form the basis of subsequent projects that receive external support.

Robert Cornell, Ph.D., UI assistant professor of anatomy and cell biology, will create a transgenic zebrafish that allows researchers to optimize gene correction strategies in zebrafish cells. Zebrafish are a commonly used lab animal; Cornell's study aims to develop new methods for the use of zebrafish as a tool in gene therapy research. The transgenic zebrafish will express a mutated, non-fluorescent version of green fluorescent protein in all its cells. Gene correction strategies can be tested using this model because restoration of fluorescence will indicate success.

Scott Harper, Ph.D., a UI research investigator in internal medicine, will develop gene therapy delivery methods that control the amount of RNA interference (RNAi) expressed in treated tissue. RNAi is a biological mechanism that can selectively turn off disease-causing genes and has the potential to treat many types of dominant disorders. Controlling the amount of RNAi in a diseased tissue will help prevent problems caused by excess amounts of RNAi.

Anton McCaffrey, Ph.D., UI assistant professor of internal medicine, will investigate a new approach for treating hepatitis B infection, which the World Health Organization lists as the ninth leading cause of death worldwide. McCaffrey and his team will design a hybrid protein to seek out and break up a specific type of viral DNA that allows hepatitis B virus to lurk within cells and cause reinfection.

Kevin Rice, Ph.D., professor of medicinal and natural products chemistry in the UI College of Pharmacy, will study a new way of delivering genes into cell nuclei without using viral vectors (stripped-down viruses used to transport corrective genes into cells). Rice and his team will develop short peptides designed to target genes to the cell nucleus. They hope to optimize this system to efficiently target muscle cells by direct injection or airway cells by jet nebulizer. The researchers will use animal models of hemophilia and cystic fibrosis to test the ability of the labeling peptides to deliver therapeutic proteins for these diseases.

Jeanne Snyder, Ph.D., UI professor of anatomy and cell biology, will investigate whether a lung protein called surfactant protein D (SP-D) plays an important role in lung disease caused by cystic fibrosis (CF). People with CF have much lower levels of SP-D in their lungs than people without the disease. People with CF also appear to have an abnormal innate immune system, which leaves the lungs less able to clear bacterial infections. Snyder's team will examine how SP-D affects the innate immune system in the lungs of genetically altered and normal mice to determine if different amounts of SP-D can affect the severity of lung disease caused by CF.

Shaf Keshavjee, M.D., senior scientist in the Toronto General Hospital Research Institute at the University of Toronto, will study the use of gene therapy to repair donor lungs for transplantation to improve their function. Success in this project has the potential to increase the number of lungs suitable for transplantation.

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

STORY SOURCE: University of Iowa Health Science Relations, 5139 Westlawn, Iowa City, Iowa 52242-1178

MEDIA CONTACT: David Pedersen, 319-335-8032, Writer: Jennifer Brown