Scientists from the University of North Carolina at Chapel Hill School of Medicine may have just happened upon the most efficient way to deliver a corrected gene to lung cells derived from cystic fibrosis patients. This new finding renews hope that gene therapy for CF lung disease could be a successful future treatment.
Scientists have worked for 20 years to perfect gene therapy for the treatment of cystic fibrosis, which causes the body to produce dehydrated, thicker-than-normal mucus that clogs the lungs and leads to life threatening infections.
In the new study, scientists used parainfluenza virus, one of the viruses that causes common colds, and found that delivery of a corrected version of the CFTR gene to 25 percent of cells grown in a tissue culture model that resembles the lining of the human airways was sufficient to restore normal function back to the tissue.
"This is the first demonstration in which we've been able to execute delivery in an efficient manner," said Ray Pickles, Ph.D., associate professor of microbiology and immunology at the UNC Cystic Fibrosis Research and Treatment Center.
"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.
"We discovered that if you take a virus that has evolved to infect the human airways, and you engineer a normal CFTR gene into it, you can use this virus to correct all of the hallmark CF features in the model system that we used," Pickles added.
For instance, the experiment improved the cells' ability to hydrate and transport mucus secretions.
The study has been published in the July 21 issue of the journal PLoS Biology.