Featured Research

from universities, journals, and other organizations

Common Cold Virus Efficiently Delivers Corrected Gene To Cystic Fibrosis Cells

Date:
July 22, 2009
Source:
University of North Carolina School of Medicine
Summary:
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. Now scientists have found what may be the most efficient way to deliver a corrected gene to lung cells collected from cystic fibrosis patients.

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.

Related Articles


Now University of North Carolina at Chapel Hill School of Medicine scientists have found what may be the most efficient way to deliver a corrected gene to lung cells collected from cystic fibrosis patients. They also showed that it may take this high level of efficiency for cystic fibrosis (CF) patients to see any benefit from gene therapy.

Using parainfluenza virus, one of the viruses that causes common colds, the UNC scientists 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 said. For instance, the experiment improved the cells' ability to hydrate and transport mucus secretions.

Now the researchers must work to ensure the safety of the delivery system. In a pleasant surprise, simply adding the CFTR gene to the virus significantly attenuated it, potentially reducing its ability to cause inflammation. But the scientists may need to alter the virus further.

"We haven't generated a vector that we can go out and give to patients now," Pickles said, "but these studies continue to convince us that a gene replacement therapy for CF patients will some day be available in the future."

In addition to Pickles, UNC co-authors are Liqun Zhang Ph.D, research associate, CF Center; Brian Button Ph.D., assistant professor, CF Center; Sherif E. Gabriel Ph.D., associate professor, pediatrics); Susan Burkett, research analyst, CF Center; Yu Yan, research specialist, CF Center; Yan Li Dang, research specialist, CF Center; Tristan McKay Ph.D., postdoctoral fellow, CF Center; and Richard C. Boucher M.D., Kenan Professor of Medicine, director, CF Center.

Other co-authors are April Mengos of the Mayo Clinic College of Medicine, as well as Mario H. Skiadopoulos, Ph.D., Leatrice N. Vogel and Peter L. Collins Ph.D., all of the National Institute of Allergy and Infectious Diseases, National Institutes of Health.

The research was funded by the National Institutes of Health and the Cystic Fibrosis Foundation.


Story Source:

The above story is based on materials provided by University of North Carolina School of Medicine. Note: Materials may be edited for content and length.


Journal Reference:

  1. Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, et al. CFTR Delivery to 25% of Surface Epithelial Cells Restores Normal Rates of Mucus Transport to Human Cystic Fibrosis Airway Epithelium. PLoS Biology, July 21, 2009; 7(7): e1000155 DOI: 10.1371/journal.pbio.1000155

Cite This Page:

University of North Carolina School of Medicine. "Common Cold Virus Efficiently Delivers Corrected Gene To Cystic Fibrosis Cells." ScienceDaily. ScienceDaily, 22 July 2009. <www.sciencedaily.com/releases/2009/07/090720202603.htm>.
University of North Carolina School of Medicine. (2009, July 22). Common Cold Virus Efficiently Delivers Corrected Gene To Cystic Fibrosis Cells. ScienceDaily. Retrieved March 5, 2015 from www.sciencedaily.com/releases/2009/07/090720202603.htm
University of North Carolina School of Medicine. "Common Cold Virus Efficiently Delivers Corrected Gene To Cystic Fibrosis Cells." ScienceDaily. www.sciencedaily.com/releases/2009/07/090720202603.htm (accessed March 5, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Thursday, March 5, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Bupa Eyes India Healthcare Opportunities

Bupa Eyes India Healthcare Opportunities

Reuters - Business Video Online (Mar. 5, 2015) Bupa is hoping to expand in India&apos;s fast-growing health insurance market, once a rule change on foreign investment is implemented. The British private healthcare group&apos;s CEO tells Grace Pascoe why it&apos;s so keen on the new opportunity. Video provided by Reuters
Powered by NewsLook.com
Doctor in Your Pocket Is Getting Smarter

Doctor in Your Pocket Is Getting Smarter

Reuters - Business Video Online (Mar. 5, 2015) Mobile apps are turning smartphones into a personal doctors, with users able to measure heart rate, blood pressure and even blood sugar. But will it change our behaviour? Ivor Bennett reports from the Mobile World Congress in Barcelona. Video provided by Reuters
Powered by NewsLook.com
AbbVie Inks $21B Deal To Buy Cancer Drugmaker Pharmacyclics

AbbVie Inks $21B Deal To Buy Cancer Drugmaker Pharmacyclics

Newsy (Mar. 5, 2015) AbbVie announced Wednesday it will buy cancer drugmaker Pharmacyclics in a $21 billion deal. Video provided by Newsy
Powered by NewsLook.com
Toddlers Drinking Coffee? Why You Shouldn't Share Your Joe

Toddlers Drinking Coffee? Why You Shouldn't Share Your Joe

Newsy (Mar. 5, 2015) A survey of Boston mothers and toddlers found that 15 percent of two-year-olds drink coffee and 2.5 percent of 1-year-olds. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins