Featured Research

from universities, journals, and other organizations

New RNA Repair May Lead To More Successful Gene Therapy

Date:
May 1, 1998
Source:
University Of North Carolina At Chapel Hill
Summary:
A possible new form of gene therapy designed to mask genetic mutations - instead of cutting away and replacing them -- has been developed by scientists at the University of North Carolina at Chapel Hill School of Medicine and Bern University in Switzerland.

CHAPEL HILL - A possible new form of gene therapy designed to mask genetic mutations - instead of cutting away and replacing them -- has been developed by scientists at the University of North Carolina at Chapel Hill School of Medicine and Bern University in Switzerland.

The technique, so far limited to laboratory cell cultures, involves using small RNA molecules to block defective processing, or splicing, inside cell nuclei of a messenger RNA that codes for a blood protein known as beta-globin.

Since the short RNA fragments block the faulty processing sites, cells' splicing machinery can only use functional, non-mutated locations, researchers say. What results is steady production of healthy "messengers," which then relay accurate genetic instructions into cell cytoplasm where normal proteins assemble.

A report on the research appears in the April 28 issue of the Proceedings of the National Academy of Sciences. Authors include pharmacology doctoral student Linda Gorman and Dr. Ryszard Kole, professor of pharmacology at UNC-CH.

"This work offers real hope that one day we will be able to cure - not just treat -- beta thalassemia, an inherited deficiency of hemoglobin, the essential protein that carries oxygen and gives blood its red color," said Kole, a member of the UNC Lineberger Comprehensive Cancer Center. "We are still a few steps away from trying this in patients, but it is quite promising."

Like sending imperfect plans to a factory, errors in messenger RNA production result in defective or inadequate protein production, he said. In severe cases of untreated beta thalassemia, those errors lead to acute anemia and death at a young age.

"The advantage of our novel way to block the incorrect splice sites is that the chances of doing something inappropriate to genes are minimal," Kole said. "A limitation would be that you can't just cut out the whole gene and put in the correct one. You have to know exactly what the specific mutation is and target it."

In their work, Gorman, Kole and collaborators introduced modified RNA molecules into cells containing mutated genes that cause beta thalassemia, which is common in Cyprus, the Middle East and Southeast Asia. They modified the molecules by incorporating into them sections of RNA complementary - something like a mirror image - to the defective sites.

"Adding these modified molecules led to increased levels, about 65 percent, of correctly spliced messenger RNA that carried the code for globin, a sub-unit of hemoglobin," Kole said. "While this research demonstrates that such molecules can be permanently established in cultured cells, the ultimate aim is to incorporate such particles into patients' bone marrow where red blood cells, which carry hemoglobin throughout the body, are produced."

The new gene therapy technique might work with many other genetic illnesses as well, he said.

Co-authors of the report are Drs. Victoria Emerick of SmithKline Beecham in Collegetown, Pa., and Drs. Daniel Suter and Daniel Schumperli of Bern University's Zoological Institute.

The National Institutes of Health, the Roche Research Foundation and the Swiss National Science Foundation supported the research.


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. "New RNA Repair May Lead To More Successful Gene Therapy." ScienceDaily. ScienceDaily, 1 May 1998. <www.sciencedaily.com/releases/1998/05/980501083313.htm>.
University Of North Carolina At Chapel Hill. (1998, May 1). New RNA Repair May Lead To More Successful Gene Therapy. ScienceDaily. Retrieved October 20, 2014 from www.sciencedaily.com/releases/1998/05/980501083313.htm
University Of North Carolina At Chapel Hill. "New RNA Repair May Lead To More Successful Gene Therapy." ScienceDaily. www.sciencedaily.com/releases/1998/05/980501083313.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

How Nigeria Beat Its Ebola Outbreak

How Nigeria Beat Its Ebola Outbreak

Newsy (Oct. 20, 2014) The World Health Organization has declared Nigeria free of Ebola. Health experts credit a bit of luck and the government's initial response. Video provided by Newsy
Powered by NewsLook.com
Another Study Suggests Viagra Is Good For The Heart

Another Study Suggests Viagra Is Good For The Heart

Newsy (Oct. 20, 2014) An ingredient in erectile-dysfunction medications such as Viagra could improve heart function. Perhaps not surprising, given Viagra's history. Video provided by Newsy
Powered by NewsLook.com
Ebola Worries End for Dozens on U.S. Watch Lists

Ebola Worries End for Dozens on U.S. Watch Lists

Reuters - US Online Video (Oct. 20, 2014) Forty-three people who had contact with Thomas Eric Duncan, the first person diagnosed with Ebola in the U.S., were cleared overnight of twice-daily monitoring after 21 days of showing no symptoms. Rough Cut (no reporter narration). Video provided by Reuters
Powered by NewsLook.com
Beijing Marathon Runners Brave Hazardous Air Pollution

Beijing Marathon Runners Brave Hazardous Air Pollution

AFP (Oct. 19, 2014) Tens of thousands of runners battled thick smog at the Beijing Marathon on Sunday, with some donning masks as the levels of PM2.5 small pollutant particles soared to 16 times the maximum recommended level. Duration: 00:54 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