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:9/19/2014)... that legume plants regulate their symbiotic relationship with ... transmitted through the plant structure from leaves into ... nodules in the roots. This collaborative study was ... Basic Biology, the Graduate University for Advanced Studies ... Science in Japan. , Legumes, an important ...
(Date:9/18/2014)... in the primitive brainstem has revealed how we fall ... of Medicine and the University at Buffalo School of ... "sleep node" identified in the mammalian brain whose activity ... deep sleep. , Published online in August in ... of all of the brain,s sleep-promoting activity originates from ...
(Date:9/18/2014)... can operate inside the bore of an MRI scanner ... research partnership program at Brigham and Women,s Hospital in ... in conjunction with real-time MRI images, can make prostate ... discomforting for the patient. The novel system also has ... precision. , Developed by a team of robotics engineers ...
Breaking Biology News(10 mins):For legume plants, a new route from shoot to root 2No sedative necessary: Scientists discover new 'sleep node' in the brain 2Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 2Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 3Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 4Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 5
... A new report published by an independent global ... system needs an immediate transformation to meet current and ... The Commission on Sustainable Agriculture and Climate Change today ... policy responses to the global challenge of feeding the ...
... 2011 For the first time, researchers have ... of the enzymes involved in degrading persistent pollutants ... Professor Michel Sylvestre of Centre INRSInstitut Armand-Frappier in ... not only to help develop effective biocatalysts for ...
... (University of Turku and VTT Technical Research Centre of Finland) ... Institute for Health and Welfare) have discovered that the SHARPIN ... leading journal, the study concludes that SHARPIN regulates the movement ... cancer cells. It is likely that the discovery will also ...
Cached Biology News:Global commission delivers food security policy recommendations 2Global commission delivers food security policy recommendations 3
(Date:9/19/2014)... NC (PRWEB) September 19, 2014 ... Biobased Product Label for its Organic Cotton . ... product’s amount of renewable biobased ingredients meets or exceeds ... intermediate materials composed in whole or in significant part ... logo/label will allow our Organic cotton to be immediately ...
(Date:9/19/2014)... The new , Healthcare and Life Sciences Review ... PharmaBoardroom.com for free download , looks beyond ... economy in 2009, and the austerity measures that followed, to ... times of trouble. "Despite the crisis, ... and the general conditions to invest are favorable to the ...
(Date:9/18/2014)... DALLAS , Sept. 18, 2014 /PRNewswire-iReach/ -- ... and China Acetic Acid Industry" and "2014 Deep ... Industry" reports to its research database. ... 2014 Deep Research Report on Global and China ... research report on China ...
(Date:9/18/2014)... Texas (PRWEB) September 18, 2014 ... Chancellor John Sharp, Texas A&M Health Science Center ... U.S. Department of Health and Human Services (HHS), ... dedicated a national pandemic influenza vaccine manufacturing facility ... as an anchor for the Texas A&M Biocorridor ...
Breaking Biology Technology:Barnhardt Manufacturing Company Earns USDA Certified Biobased Product Certification and Label for Organic Cotton 2PharmaBoardroom Releases New Spain Pharmaceuticals Report 2PharmaBoardroom Releases New Spain Pharmaceuticals Report 3Acetic Acid Market & Sodium Stannate Industry (China, Global) Research Report 2Acetic Acid Market & Sodium Stannate Industry (China, Global) Research Report 3Acetic Acid Market & Sodium Stannate Industry (China, Global) Research Report 4Texas A&M Dedicates National Pandemic Influenza Vaccine Manufacturing Facility, on Track for 2016 Start-Up Phase 2Texas A&M Dedicates National Pandemic Influenza Vaccine Manufacturing Facility, on Track for 2016 Start-Up Phase 3Texas A&M Dedicates National Pandemic Influenza Vaccine Manufacturing Facility, on Track for 2016 Start-Up Phase 4
... Advanced Biofuels, LLC, a joint venture between BP ... into collaboration with Fagen, Inc. for the introduction ... is the world,s most experienced biofuels engineering, procurement ... than any other company.  They are the largest, ...
... Exciting new data presented today at the ... role in the development of diabetes and non-alcoholic fatty ... . Though an early stage animal model, the French ... with gut microbiota transplantation - the engrafting of new ...
... , Luis Montaner from the Wistar Institute, Philadelphia, USA ... model to a cohort of HIV-infected patients on antiretroviral ... be used to optimize laboratory capacity in settings where ... is not intended to replace CD4 count testing or ...
Cached Biology Technology:Butamax™ and Fagen Announce Collaboration to Retrofit Ethanol Plants for Biobutanol Production 2Gut Microbiota Transplantation may Prevent Development of Diabetes and Fatty Liver Disease 2Gut Microbiota Transplantation may Prevent Development of Diabetes and Fatty Liver Disease 3
... siSolutions siRNA Custom Synthesis ... effective duplex RNA to knockdown ... 21-mers (19 RNA base pairs ... recently-published 27-mers, or other lengths ...
EDG-1 Western blot control Source: Cell Lysate Application: Western Blotting Control...
Hybridization cassette for one slide...
Request Info...
Biology Products: