Featured Research

from universities, journals, and other organizations

Gene Therapy Hold Promise For Sickle Cell Disease And Beta Thalassemia

Date:
November 4, 1997
Source:
Columbia University College Of Physicians And Surgeons
Summary:
Researchers from Columbia University College of Physicians & Surgeons have demonstrated the long-term transfer and high level long-term expression of the normal human beta globin gene in an animal model for the first time. The study may lead to gene therapy for the treatment of sickle cell disease and beta thalassemia, a related disorder.

Researchers from Columbia University College of Physicians & Surgeons have demonstrated the long-term transfer and high level long-term expression of the normal human beta globin gene in an animal model for the first time. The study, published in the Nov. 1 issue of Blood, may lead to gene therapy for the treatment of sickle cell disease and beta thalassemia, a related disorder.

Senior author Arthur Bank, M.D., FACP, professor of medicine and genetics & development and director of the division of hematology at Columbia University, and colleagues put a human beta globin gene into a safe retrovirus and added the virus to mice bone marrow cells in vivo. The modified cells were then transplanted into mice. The researchers were able to detect the presence of the human beta globin gene up to eight months later. In addition, the researchers documented high levels of expression of the gene. In one mouse, 20 percent of the total beta globin it produced was from the human beta globin gene.

"If we could attain that level of normal human beta globin gene expression in a patient with sickle cell disease or beta thalassemia marrow cells, it would be enough to expect to ameliorate, if not cure, the anemia of patients with sickle cell disease and beta thalassemia," says Bank.

In sickle cell disease, the beta globin gene produces an abnormal form of hemoglobin, the oxygen-carrying molecule in the blood. The damage causes red blood cells to distort and alter their shape. In beta thalassemia, the gene produces inadequate levels of hemoglobin. Theoretically, gene therapy that inserts a normal form of the beta globin gene into the bone marrow cells of patients could cure these diseases.

"For more than 10 years researchers have been looking for a stable delivery system to transfer and express the human beta globin gene for extended periods of time. This is the first time researchers safely and efficiently transferred the normal globin and ensured that the gene produced normal levels of its protein over long periods of time," says Dr. Bank

Researchers are investigating better gene transfer systems in mouse models of sickle cell disease and beta thalassemia and developing better ways to transfer retroviruses into human hematopoietic stem cells.

In addition to Dr. Bank, the authors of the paper were Dr. Harry Raftopoulos and Maureen Ward of Columbia Univertsity MC, and Dr. Philippe Leboulch of Harvard Medical School and the Massachusetts Institute of Technology.

The study was funded by the National Institutes of Health; the Cooley's Anemia Foundation; and the Ahepa Anemia Foundation.


Story Source:

The above story is based on materials provided by Columbia University College Of Physicians And Surgeons. Note: Materials may be edited for content and length.


Cite This Page:

Columbia University College Of Physicians And Surgeons. "Gene Therapy Hold Promise For Sickle Cell Disease And Beta Thalassemia." ScienceDaily. ScienceDaily, 4 November 1997. <www.sciencedaily.com/releases/1997/11/971104063108.htm>.
Columbia University College Of Physicians And Surgeons. (1997, November 4). Gene Therapy Hold Promise For Sickle Cell Disease And Beta Thalassemia. ScienceDaily. Retrieved July 24, 2014 from www.sciencedaily.com/releases/1997/11/971104063108.htm
Columbia University College Of Physicians And Surgeons. "Gene Therapy Hold Promise For Sickle Cell Disease And Beta Thalassemia." ScienceDaily. www.sciencedaily.com/releases/1997/11/971104063108.htm (accessed July 24, 2014).

Share This




More Health & Medicine News

Thursday, July 24, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

New Painkiller Designed To Discourage Abuse: Will It Work?

New Painkiller Designed To Discourage Abuse: Will It Work?

Newsy (July 24, 2014) The FDA approved Targiniq ER on Wednesday, a painkiller designed to keep users from abusing it. Like any new medication, however, it has doubters. Video provided by Newsy
Powered by NewsLook.com
Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Doctor At Forefront Of Fighting Ebola Outbreak Gets Ebola

Newsy (July 24, 2014) Sheik Umar Khan has treated many of the people infected in the Ebola outbreak, and now he's become one of them. Video provided by Newsy
Powered by NewsLook.com
Condemned Man's US Execution Takes Nearly Two Hours

Condemned Man's US Execution Takes Nearly Two Hours

AFP (July 24, 2014) America's death penalty debate raged Thursday after it took nearly two hours for Arizona to execute a prisoner who lost a Supreme Court battle challenging the experimental lethal drug cocktail. Duration: 00:55 Video provided by AFP
Powered by NewsLook.com
China's Ageing Millions Look Forward to Bleak Future

China's Ageing Millions Look Forward to Bleak Future

AFP (July 24, 2014) China's elderly population is expanding so quickly that children struggle to look after them, pushing them to do something unexpected in Chinese society- move their parents into a nursing home. Duration: 02:07 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:
from the past week

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