Featured Research

from universities, journals, and other organizations

Jefferson Scientists Further Develop Gene Repair Technique

Date:
June 15, 1999
Source:
Thomas Jefferson University
Summary:
Molecular geneticists at Jefferson Medical College are inching closer to using a novel gene repair technique to treat diseases such as cystic fibrosis and sickle cell anemia. While the work remains in the early stages, the scientists are hopeful that their technique will eventually be able to correct the genetic defect responsible for more than 70 percent of cystic fibrosis cases.

Researchers, in creating new gene repair molecules, are edging closer to potential applications

Related Articles


Molecular geneticists at Jefferson Medical College are inching closer to using a novel gene repair technique to treat diseases such as cystic fibrosis and sickle cell anemia. While the work remains in the early stages, the scientists are hopeful that their technique will eventually be able to correct the genetic defect responsible for more than 70 percent of cystic fibrosis cases.

Several years ago, Eric Kmiec, Ph.D., associate professor of microbiology and immunology at Thomas Jefferson University in Philadelphia and a member of Jefferson’s Kimmel Cancer Center, developed a technique to repair genes with a single mutation. It involved synthesizing a small molecule termed a chimera, consisting of DNA interspersed with small amounts of RNA, that has the capability to find and attach itself to a certain part of a gene. The small genetic vehicle is designed to trigger the cell’s normal DNA repair system into action as well. The repair mechanism scans the DNA looking for any mismatches or two strands of DNA that don’t seem in sync. When it finds a mismatch, it replaces one of the chemical bases with one that fits better. The scientists, then, can use this natural repair system to correct a bad mutation.

Now, he and his colleagues at the Jefferson Center for Biomedical Research have extended the limits of the current technology. Dr. Kmiec presents his latest research June 13 at the meeting of the American Society of Gene Therapy in Washington, D.C.

Dr. Kmiec’s team was able to correct both point and frameshift genetic mutations – two different types of single gene base mutations – of the globin gene involved in manufacturing hemoglobin and show the changes were inheritable. He also used the technique to fix copies of the green fluorescent protein gene, which contained one- or two-base deletions. His team has "learned more about how the molecule works," he says, which will "set the stage for direct work in cystic fibrosis and other diseases that are caused by deletions of large parts of DNA." A three-base pair deletion in a gene causes more than 70 percent of cystic fibrosis cases.

Dr. Kmiec had shown previously that the new gene repair technology may hold promise as a treatment for sickle cell anemia and other diseases by correcting the DNA mutation from which they arise.

His aim is to create a "new generation of molecules" to treat disease more effectively. Though the gene correction may occur in only 5 percent of cells, for some diseases, he suggests, this may be enough to help alleviate symptoms, if not cure the disease.

"What’s exciting," he says, "is that we have some newer and better chimera structures that improve gene correction frequency and will move us toward cystic fibrosis and sickle cell anemia applications."


Story Source:

The above story is based on materials provided by Thomas Jefferson University. Note: Materials may be edited for content and length.


Cite This Page:

Thomas Jefferson University. "Jefferson Scientists Further Develop Gene Repair Technique." ScienceDaily. ScienceDaily, 15 June 1999. <www.sciencedaily.com/releases/1999/06/990615080802.htm>.
Thomas Jefferson University. (1999, June 15). Jefferson Scientists Further Develop Gene Repair Technique. ScienceDaily. Retrieved October 31, 2014 from www.sciencedaily.com/releases/1999/06/990615080802.htm
Thomas Jefferson University. "Jefferson Scientists Further Develop Gene Repair Technique." ScienceDaily. www.sciencedaily.com/releases/1999/06/990615080802.htm (accessed October 31, 2014).

Share This



More Health & Medicine News

Friday, October 31, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

U.S. Ebola Response Measures Demonstrated

U.S. Ebola Response Measures Demonstrated

AP (Oct. 31, 2014) Officials in the Washington area showed off Ebola response measures being taken at Dulles International Airport and the National Institutes of Health. (Oct. 31) Video provided by AP
Powered by NewsLook.com
Fauci Says Ebola Risk in US "essentially Zero"

Fauci Says Ebola Risk in US "essentially Zero"

AP (Oct. 30, 2014) NIAID Director Anthony Fauci said the risk of Ebola becoming an epidemic in the U.S. is essentially zero Thursday at the Washington Ideas Forum. He also said an Ebola vaccine will be tested in West Africa in the next few months. (Oct. 30) Video provided by AP
Powered by NewsLook.com
Nurse Defies Ebola Quarantine With Bike Ride

Nurse Defies Ebola Quarantine With Bike Ride

AP (Oct. 30, 2014) A nurse who vowed to defy Maine's voluntary quarantine for health care workers who treated Ebola patients followed through on her promise Thursday, leaving her home for an hour-long bike ride. (Oct. 30) Video provided by AP
Powered by NewsLook.com
Pot-Infused Edibles Raise Concerns in Colorado

Pot-Infused Edibles Raise Concerns in Colorado

AFP (Oct. 30, 2014) Colorado may have legalized marijuana for recreational use, but the debate around the decision still continues, with a recent - failed - attempt to ban cannabis-infused edibles. Duration: 01:53 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