Navigation Links
Gene therapy corrects sickle cell disease in laboratory study
Date:12/3/2008

Using a harmless virus to insert a corrective gene into mouse blood cells, scientists at St. Jude Children's Research Hospital have alleviated sickle cell disease pathology. In their studies, the researchers found that the treated mice showed essentially no difference from normal mice. Although the scientists caution that applying the gene therapy to humans presents significant technical obstacles, they believe that the new therapy will become an important treatment for the disease.

Sickle cell disease, which affects millions of people worldwide, arises because of a tiny genetic defect in the gene for beta-globin, a protein component of hemoglobin. This defect causes hemoglobin-containing red blood cells to tend to deform, clump and break apart. The resulting clogged blood vessels can lead to cognitive dysfunction by causing small strokes in the brain and cause damage to kidneys, liver, spleen and lungs. The only permanent cure for the disease is a bone marrow transplant to give recipients blood-forming cells that will form normal beta-globin. However, such transplants are rare because of the lack of compatible donors.

Researchers have long known that symptoms of the disease could be alleviated by persistence in the blood of an immature fetal form of hemoglobin in red blood cells. This immature hemoglobin, which usually disappears after birth, does not contain beta-globin, but another form called gamma-globin. St. Jude researchers had found that treating patients with the drug hydroxyurea encourages the formation of fetal hemoglobin and alleviates disease symptoms.

"While this is a very useful treatment for the disease, our studies indicated that it might be possible to cure the disorder if we could use gene transfer to permanently increase fetal hemoglobin levels," said Derek Persons, M.D., Ph.D., assistant member in the St. Jude Department of Hematology.

He and his colleagues developed a technique to insert the gene for gam
'/>"/>

Contact: Summer Freeman
summer.freeman@stjude.org
901-595-3061
St. Jude Children's Research Hospital
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. A new radiation therapy treatment developed for head and neck cancer patients
2. St. Jude finds factors that accelerate resistance to targeted therapy in lymphoblastic leukemia
3. UC health news: molecular pathway may predict chemotherapy effectiveness
4. MIT works toward safer gene therapy
5. Intravenous gene therapy protects normal tissue of mice during whole-body radiation
6. Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart
7. Fourth Annual International Conference on Cell Therapy for Cardiovascular Diseases
8. Safe and effective therapy discovered for patients with protein-losing enteropathy
9. Ireland Cancer Center researchers advance stem cell gene therapy
10. Dolphin therapy a dangerous fad, Emory researchers warn
11. Cancer and arthritis therapy may be promising treatment for diabetes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/11/2014)... CHICAGO , Nov. 11, 2014  Forensicon, Inc., ... eDiscovery firm, is pleased to announce the promotion of ... to Director of Digital Forensics. In Schiff,s new role ... out the team of digital forensics examiners and provide ... Photo - http://photos.prnewswire.com/prnh/20141110/157719 ...
(Date:11/7/2014)... Researchers led by Dr. Debra Auguste, associate professor, ... at The City College of New York, have ... treatment for one of the most aggressive forms ... (TNBCs) have a high mortality rate owing to ... effective therapeutic options. However, Professor Auguste,s team, discovered ...
(Date:11/7/2014)... , November 6, 2014 Leading ... for better mobile security revolutionizing online transactions.  Companies in focus ... Holdings Ltd. (NYSE: BABA ), Google Inc. (NASDAQ: ... ), eBay Inc. (NASDAQ: EBAY ) and MasterCard ... NXTD and NXTDW), a biometric authentication company focused on ...
Breaking Biology News(10 mins):Yaniv Schiff Promoted to Director of Digital Forensics at Forensicon, Inc. 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 2Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 3Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 4Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 5Universal Authentication Methods Provide More Secure Operations for Biometrics Devices - Company Joins FIDO Alliance for Consumer Protection 6
... molecules of membranes, also known as phospholipids, are composed of ... The molecules form a bilayer in the membrane, with all ... hanging in a zip-like interlay position. Biomembranes are ... cell is constantly creating new phospholipids that have to align ...
... JUPITER, FL, January 29, 2013 While the wooly musk ox ... it hot, or at least warm. In fact, their preferred optimum ... Scientists have known that a type of brain cell circuit helps ... their temperature preferences. What has been a mystery is whether or ...
... Trees in the continental U.S. could send out new spring ... than they did before global temperatures started to rise, according ... changes could lead to changes in the composition of northeastern ... up carbon dioxide. Trees play an important role in ...
Cached Biology News:Pathway for membrane building blocks 2Scripps Research Institute study shows how brain cells shape temperature preferences 2Spring may come earlier to North American forests 2Spring may come earlier to North American forests 3
(Date:11/21/2014)... , Nov. 21, 2014   TRU-D SmartUVC LLC ... their superbug-killing UVC automated disinfection robot, TRU-D SmartUVC, at FIS ... from Nov. 23 to 26 at Stand 23. ... United Kingdom,s largest infection-related event of ... societies to one of the leading events of its type ...
(Date:11/21/2014)... 2014 Offering a complete line of ... Balance promotion . The Sartorius Entris Balance is the ... accuracy, and easy-to-maintain balance entry level balance. , The ... designed to help customer’s bridge the gap between a ... is very durable. Sartorius is the second biggest balance ...
(Date:11/21/2014)... November 20, 2014 The ... policy research organization representing leading California academic institutions, ... PwC US today released a report ... sciences industry growth. The trend shows a 4 ... 2015 California Biomedical Industry Report indicates that, ...
(Date:11/21/2014)... York, NY (PRWEB) November 20, 2014 ... is now available as an open access journal featuring ... with infrared fluorescent proteins to preserve photoresponsiveness in the ... Two-color fluorescent in situ hybridization using chromogenic substrates ... chips for DNA, RNA, and protein electrophoresis , ...
Breaking Biology Technology:TRU-D SmartUVC to Showcase Superbug-Killing Robot at FIS 2014 2TRU-D SmartUVC to Showcase Superbug-Killing Robot at FIS 2014 3Pipette.com Announces an Extension on the Sartorius Entris Balance Promotion to the End of 2014 2California Healthcare Institute and PwC Report Boom in California’s Biomedical Industry 2California Healthcare Institute and PwC Report Boom in California’s Biomedical Industry 3California Healthcare Institute and PwC Report Boom in California’s Biomedical Industry 4BioTechniques: The International Journal of Life Science Methods Announces the Publication of the November 2014 Issue—Available in Print and Online 2BioTechniques: The International Journal of Life Science Methods Announces the Publication of the November 2014 Issue—Available in Print and Online 3
... government-mandated change as a result of the Sarbanes-Oxley ... reporting and companies to continually evaluate their financial ... as to the technology requirements and information technology ... any technology; however, it is difficult to envision ...
... Wednesday it will support American Musical Supplys (AMS) ... chain execution solutions. AMS , a supplier ... distribution network next year and develop business-to-business sales ... vision products. , ,AMS was driven to ...
... Act went info effect Jan. 1 and, although its enforceability ... to come. , ,CAN-SPAM (controlling the assault of non-solicited pornography ... reduce the flow of unsolicited e-mail (SPAM). According to the ... , , Clearly label commercial e-mail as advertising , Use ...
Cached Biology Technology:Technology Implications of Sarbanes-Oxley 2Technology Implications of Sarbanes-Oxley 3CAN-SPAM: Wheres the Meat? 2
... Zyagen tissue microarrays are derived from frozen ... rigorous quality control procedures to ensure the ... manually assembled in one block, sectioned at ... coated slides. Sections are large (3-5mm in ...
... Opticon real-time PCR detection system uses a ... (450-495 nm) and a photomultiplier tube for ... is built on an DNA Engine thermal ... feature. This item includes the thermal cycler, ...
... Precision-designed for Optimal Microdissection , CapSure ... the precise and rapid extraction of populations ... cytology samples with Laser Capture Microdissection., ... Downstream Molecular Analysis , All CapSure LCM ...
... are highly sensitive laser confocal systems designed ... advanced scanners that meet your expectations, differentiating ... and optimizing the signal on the bottom ... the range for increased sensitivity. Results with ...
Biology Products: