Featured Research

from universities, journals, and other organizations

Pinpoint Targeting Promises Improved Success In Gene Therapy

Date:
August 18, 1999
Source:
University Of North Carolina At Chapel Hill
Summary:
Research on a rare inherited blood disorder known as Glanzmann's disease shows for the first time that it's possible to target specific types of cells during gene therapy and avoid the less effective -- and possibly more dangerous -- "shotgun" approach now used, according to a new University of North Carolina at Chapel Hill School of Medicine study.

CHAPEL HILL - Research on a rare inherited blood disorder known as Glanzmann's disease shows for the first time that it's possible to target specific types of cells during gene therapy and avoid the less effective -- and possibly more dangerous -- "shotgun" approach now used, according to a new University of North Carolina at Chapel Hill School of Medicine study.

With more work, the method could help treat a host of gene-related illnesses including various cancers, UNC-CH scientists say.

"Using techniques first developed by other investigators, we created a way of targeting the expression, or production, of genetic material in blood platelets without affecting red or white blood cells at the same time," said Dr. Gilbert C. White II, professor of medicine and director of UNC-CH's Center for Thrombosis and Hemostasis. "This offers great promise for improved treatment of inherited blood disorders such as hemophilia and conceptual support for using the approach for non-inherited illnesses such as cancer.

"For example, if you wanted to get poison genes into cancer cells but not into other healthy cells, one could attach the poison gene to specific promoters -- or pieces of DNA - that function only in cancer cells and not in others."

A report on the study appears in the Aug. 17 issue of the Proceedings of the National Academy of Sciences. Besides White, authors are Drs. David A. Wilcox, a former UNC-CH postdoctoral fellow now at the Medical College of Wisconsin; John C. Olsen, research assistant professor of medicine at UNC-CH; and Lori Ishizawa and Michael Griffith of Nexell Therapeutics Inc. of Irvine, Calif.

Wilcox said he and his colleagues concentrated on correcting primitive stem cells - sometimes considered "parent" cells -- capable of developing into more specialized large cells. Those younger monster cells, called megakaryocytes, form in bone marrow and eventually shatter into at least hundreds of particles that become blood platelets. The body uses platelets, which float around in the bloodstream, to plug wound sites to stop bleeding and heal cuts and other wounds.

The scientists focused on Glanzmann's disease - which, because of a missing protein, prevents platelets from functioning properly -- as a model system for their work. In the laboratory, they succeeded in correcting the protein defect in the parent cells by inserting healthy genes into DNA with the help of a harmless virus that penetrated the cell walls.

"This work is novel to gene therapy because the method successfully uses a promoter that targets a protein that was missing or defective in megakaryocytes and not in other cell types," Wilcox said. Conceivably, all sorts of healthy replacement genes or even drugs could be attached to identical or similar promoters and function as well as they did in platelets but without affecting unrelated cells and cell functions, he said. Extensive testing of "daughter" cells such as red cells and white cells confirmed the method worked only where it was supposed to. There was no potentially hazardous effect on other cells.

"We target the parent stem cells because we want the therapy to last for a long time in humans," Wilcox said. "That wouldn't be possible if we just targeted platelets since a platelet's life span in the bloodstream averages only 21 days."

Wilcox, who called the findings "exciting and potentially very important," said more advanced studies already are under way at UNC-CH. Mouse and human cell experiments, while still in preliminary stages, have progressed well so far, he said.


Story Source:

The above story is based on materials provided by University Of North Carolina At Chapel Hill. Note: Materials may be edited for content and length.


Cite This Page:

University Of North Carolina At Chapel Hill. "Pinpoint Targeting Promises Improved Success In Gene Therapy." ScienceDaily. ScienceDaily, 18 August 1999. <www.sciencedaily.com/releases/1999/08/990818071552.htm>.
University Of North Carolina At Chapel Hill. (1999, August 18). Pinpoint Targeting Promises Improved Success In Gene Therapy. ScienceDaily. Retrieved October 20, 2014 from www.sciencedaily.com/releases/1999/08/990818071552.htm
University Of North Carolina At Chapel Hill. "Pinpoint Targeting Promises Improved Success In Gene Therapy." ScienceDaily. www.sciencedaily.com/releases/1999/08/990818071552.htm (accessed October 20, 2014).

Share This



More Health & Medicine News

Monday, October 20, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Microneedle Patch Promises Painless Pricks

Microneedle Patch Promises Painless Pricks

Reuters - Innovations Video Online (Oct. 18, 2014) Researchers at The National University of Singapore have invented a new microneedle patch that could offer a faster and less painful delivery of drugs such as insulin and painkillers. Video provided by Reuters
Powered by NewsLook.com
Raw: Nurse Nina Pham Arrives in Maryland

Raw: Nurse Nina Pham Arrives in Maryland

AP (Oct. 17, 2014) The first nurse to be diagnosed with Ebola at a Dallas hospital walked down the stairs of an executive jet into an ambulance at an airport in Frederick, Maryland, on Thursday. Pham will be treated at the National Institutes of Health. (Oct. 16) Video provided by AP
Powered by NewsLook.com
Raw: Cruise Ship Returns to US Over Ebola Fears

Raw: Cruise Ship Returns to US Over Ebola Fears

AP (Oct. 17, 2014) A Caribbean cruise ship carrying a Dallas health care worker who is being monitored for signs of the Ebola virus is heading back to Texas, US, after being refused permission to dock in Cozumel, Mexico. (Oct. 17) Video provided by AP
Powered by NewsLook.com
Spanish Govt: Four Suspected Ebola Cases in Spain Test Negative

Spanish Govt: Four Suspected Ebola Cases in Spain Test Negative

AFP (Oct. 17, 2014) All four suspected Ebola cases admitted to hospitals in Spain on Thursday have tested negative for the deadly virus in a first round of tests, the government said Friday. Duration: 00:55 Video provided by AFP
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