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PACEMAKER: Fall 2003

Deadly Ebola virus may boost cystic fibrosis gene therapy

Becky Soglin


Medical scientists seeking to develop gene therapy for patients with cystic fibrosis face a dilemma: delivering genes into airway cells is inefficient and disrupts the integrity of the cell layer.

However, University of Iowa Health Care researchers and colleagues have identified a surprising tool that may help solve the problem--a modified piece of the deadly Ebola virus. The findings appear in the Journal of Virology.

The investigators found that taking a small part of the protein coat from the Ebola virus and putting it on another modified virus creates a hybrid vehicle that can attach itself to a receptor on the top surface, or airway side, of lung cells. The genes within the vehicle can then enter the host cell, where they integrate into the host chromosomes.

Previously studied gene delivery vehicles can attach only to the bottom surface, which requires breaching the normally closed cell surface layer. The discovery has implications for treating patients with cystic fibrosis and other lung conditions where the genes needed for healthy lung function are mutated or absent.

"Most people understandably do not think of the Ebola virus in a favorable light (the virus causes a highly contagious fever and there is no known effective treatment)," said Paul McCray, M.D., lead investigator of the UI study. "However, our approach takes a small part from this bad virus and puts it in a hybrid system to get some good out of it."

Patrick Sinn, Ph.D., the paper's lead author, said a large region of the Ebola protein coat, or envelope, is deleted, so it is potentially safer to use.

Last modification date: Fri Dec 21 11:01:13 2007
URL: http://www.uihealthcare.com /news/pacemaker/2003/fall/ebola.html