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Release: April 27, 1999

UI receives $7.1 million NIH grant for continued cystic fibrosis gene therapy research

IOWA CITY, Iowa -- The University of Iowa College of Medicine has received a $7.1 million, five-year grant from the National Institutes of Health (NIH) to continue the study of gene therapy for cystic fibrosis.

The grant is a funding renewal from the NIH's National Heart, Lung and Blood Institute. The institute originally awarded $4.3 million of support to the UI five years ago. Renowned cystic fibrosis investigator Michael Welsh, M.D., professor of internal medicine and physiology and biophysics, and a Howard Hughes Medical Institute investigator, will continue to lead the research team, which includes five other investigators from four departments.

Cystic fibrosis is the most common fatal inherited disease among Caucasians of Northern European descent and occurs in about one in every 2,000 births. Cystic fibrosis involves several organ systems, including the lungs, sweat gland ducts and pancreas. Abnormally thick mucus builds up in the lungs of people with cystic fibrosis and can lead to inflammation and tissue damage. Although advances in treating complications of the disease have increased the average survival rate to age 21, there is no cure.

Welsh has spent much of his career at the UI studying the genetic causes of cystic fibrosis in hopes of developing strategies to treat or possibly cure the disease. One promising approach, and the one for which the UI received the NIH funding, is gene therapy. A genetic flaw causes cystic fibrosis. If investigators can correct the flaw, they can perhaps cure the disease. Gene therapy involves using a vector, such as a disabled cold virus, to supply cells with healthy copies of the flawed genes.

The UI's $7.1 million NIH grant will fund three main research projects. It will also fund three core facilities that provide the investigators with reagents and model systems.

"The goal over the next five years is to better understand the barriers that limit gene transfer to the lungs and then to discover novel ways to overcome these barriers," Welsh said. "The investigators will collaborate closely to develop several new approaches for inserting a normal gene into cystic fibrosis lung cells. Ultimately, the work should lead to studies in humans."

In addition to Welsh, the other UI investigators include E. Peter Greenberg, Ph.D., professor of microbiology; Beverly Davidson, Ph.D., associate professor of internal medicine; Paul McCray, M.D., associate professor of pediatrics; Jeffrey J. Smith, M.D., associate professor of pediatrics; and Joseph Zabner, M.D., assistant professor of internal medicine.