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.

Related Articles


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 January 31, 2015 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 January 31, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Saturday, January 31, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

CDC: Get Vaccinated for Measles

CDC: Get Vaccinated for Measles

Reuters - US Online Video (Jan. 30, 2015) The CDC is urging people to get vaccinated for measles amid an outbreak that began at Disneyland and has now infected more than 90 people. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
Obama To Outline New Plan For Personalized Medicine

Obama To Outline New Plan For Personalized Medicine

Newsy (Jan. 30, 2015) President Obama is expected to speak with drugmakers Friday about his Precision Medicine Initiative first introduced last week. Video provided by Newsy
Powered by NewsLook.com
NFL Concussions Down; Still on Parents' Minds

NFL Concussions Down; Still on Parents' Minds

AP (Jan. 30, 2015) The NFL announced this week that the number of game concussions dropped by a quarter over last season. Still, the dangers of the sport still weigh on players, and parents&apos; minds. (Jan. 30) Video provided by AP
Powered by NewsLook.com
U.S. Wants to Analyze DNA from 1 Million People

U.S. Wants to Analyze DNA from 1 Million People

Reuters - US Online Video (Jan. 30, 2015) The U.S. has proposed analyzing genetic information from more than 1 million American volunteers to learn how genetic variants affect health and disease. Rough Cut (no reporter narration). Video provided by Reuters
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