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UI Researchers Find Potential Gene Therapy For Cystic Fibrosis

IOWA CITY, Iowa -- A team of University of Iowa investigators may have found a way to permanently deliver therapeutic genes to the lungs of patients with cystic fibrosis (CF), the most common fatal hereditary disease in the United States.

The research using human cells in laboratory cultures indicates that the method could be used to permanently cure CF and other genetic lung diseases, said Paul McCray, M.D., associate professor of pediatrics in the UI College of Medicine and co-principal investigator.

The findings were published in the December issue of the Journal of Virology. The other co-principal investigators of the study were Beverly L. Davidson, Ph.D., UI associate professor of internal medicine and director of the Gene Transfer Vector Core, and Guoshun Wang, Ph.D., UI research investigator in pediatrics.

According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 children and young adults in North America. The disease occurs in approximately one of every 3,300 live births and is usually diagnosed in early childhood. Reduced lung function, lung infection and liver and pancreatic disease are among the serious conditions that can reduce the life expectancy of a person with CF. The median age of survival for a person with the disease is 31 years.

CF is caused by the malfunction of an ion channel that regulates secretion of salt and water that protect the lungs. A specialized protein, known as the CF transmembrane conductance regulator (CFTR), makes the channel. The gene that codes for the CFTR protein is mutated in people with CF. McCray said the UI research project was designed to complement other CF treatments under laboratory investigation, including another UI study. Those methods involve inserting the CFTR gene into a vector--a nonviral carrier or an adenovirus--to facilitate transferring the gene into cells lining the airway.

In some cases, the mucous produced by the diseased lungs may trap the vector and prevent
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Contact: Becky Soglin
becky-soglin@uiowa.edu
319-335-6660
University of Iowa
22-Dec-1998


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