Endeavors magazine: Cold Virus Delivers CF Gene

Carolina researchers have found what may be the most efficient way to deliver gene therapy to the lung cells of cystic fibrosis patients. The researchers used a parainfluenza virus — one of the viruses that cause common colds — to deliver a corrected version of the CFTR gene to lung cells grown in a tissue culture model that resembled the lining of the human airway. They were able to deliver the gene to 25 percent of the lung cells, enough to restore normal function in the tissue.

“When you consider that in past gene-therapy studies, the targeting efficiency has been somewhere around 0.1 percent of cells, you can see this is a giant leap forward,” says microbiologist Ray Pickles.

The experiment also improved the cells’ability to hydrate and transport mucus secretions. “We haven’t generated a vector that we can go out and give to patients,” Pickles says. “But these studies continue to convince us that a gene replacement therapy for CF patients will someday be available.”

This study was published in the journal PLoS Biology on July 21, 2009. The National Institutes of Health and the Cystic Fibrosis Foundation provided funding. Ray Pickles is an associate professor in the Department of Microbiology and Immunology.