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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.
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