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:12/10/2014)... , Dec. 8, 2014 You,ve been here ... account but can,t remember your password, site key or the ... was your first grade teacher? Today, ... the app that will finally put an end to ... 1U TM . 1U leverages a user,s smartphone ...
(Date:12/10/2014)...  Wake Forest Baptist Medical Center today announced plans for ... Medicine. Funding for this $50 million capital project is ... publicly launched next summer. The medical ... series R.J. Reynolds Tobacco Company complex, adjacent to 525@vine ... with plans to be ready to welcome medical students ...
(Date:12/10/2014)... Research and Markets ( http://www.researchandmarkets.com/research/xclcvw/biometrics_market ... "Biometrics Market in Japan 2014-2018" report to ... The integration of biometrics and ... and upgradation of the driver,s license is one ... Besides the aforementioned projects, biometrics is being used ...
Breaking Biology News(10 mins):The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 2The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 3Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 2Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 3Wake Forest Baptist to Build New Medical Education Facility In Wake Forest Innovation Quarter 4Biometrics Market in Japan 2014-2018: Key Vendors are DDS, Fujitsu, Hitachi and NEC 2
... Taking aim at a leading cause of ... at the Massachusetts Biologic Laboratories (MBL) of the University of ... that neutralizes the Hepatitis C virus (HCV). The new ... infection by the virus in a pre-clinical animal model of ...
... details of a membership agreement with BioMed Central to ... researchers. Pfizer,s BioMed Central membership arrangement means that ... researcher employed or funded by Pfizer when they submit ... peer-reviewed open access journals. By funding open access publication ...
... of Guam Associate Professor Laurie Raymundo is senior editor ... and management of coral diseases, " Coral Disease Handbook: ... summarizes the most recently developed methods for studying the ... the book is a set of identification cards for ...
Cached Biology News:Novel antibody prevents infection by hepatitis C virus 2Novel antibody prevents infection by hepatitis C virus 3
(Date:12/17/2014)... , Dec. 16, 2014  Vermillion, Inc. (NASDAQ: ... on gynecologic disease, today announced the next step in ... premier bio-analytics solutions provider with the naming of current ... and Chief Executive Officer, while current Chairman of the ... to serve as Chairman. These changes are effective January ...
(Date:12/15/2014)... METTLER TOLEDO is pleased to announce that the ... PVM technology , is now available. The new ... continuously captures high-resolution images under a wide range ... a report pairing the most relevant images to ... compelling blend of high resolution images and trend ...
(Date:12/15/2014)... , Dec. 15, 2014  Ascendis Pharma ... TransCon technology to address significant unmet medical needs, ... ongoing Phase 2 pediatric study to evaluate once-weekly ... deficiency, or GHD.  This interim analysis consists of ... total enrollment in the study, completing all six ...
(Date:12/13/2014)... and TAICHUNG, Taiwan (PRWEB) December 12, 2014 ... less rosy indicators in overall global economic growth, Eugene ... optics and photonics , said in a featured talk ... on 5 December at the National Chung Hsing University ... which is being integrated into future energy supplies and ...
Breaking Biology Technology:Vermillion Names Valerie Palmieri CEO; James LaFrance to continue as Chairman of Board 2Vermillion Names Valerie Palmieri CEO; James LaFrance to continue as Chairman of Board 3Vermillion Names Valerie Palmieri CEO; James LaFrance to continue as Chairman of Board 4New Real-Time In Situ Probe-Based Video Microscope from METTLER TOLEDO 2Ascendis Pharma A/S Announces Positive Six-Month Interim Results from a Phase 2 Pediatric Study of Once-Weekly TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency 2Ascendis Pharma A/S Announces Positive Six-Month Interim Results from a Phase 2 Pediatric Study of Once-Weekly TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency 3Ascendis Pharma A/S Announces Positive Six-Month Interim Results from a Phase 2 Pediatric Study of Once-Weekly TransCon Growth Hormone for the Treatment of Growth Hormone Deficiency 4Photonics Continues Upward Trends Despite Challenges in the Global Economy, Notes SPIE CEO 2Photonics Continues Upward Trends Despite Challenges in the Global Economy, Notes SPIE CEO 3Photonics Continues Upward Trends Despite Challenges in the Global Economy, Notes SPIE CEO 4Photonics Continues Upward Trends Despite Challenges in the Global Economy, Notes SPIE CEO 5
... New System to Streamline Process, Drive Value from ... Software Inc., a leading,provider of web-based employee performance ... been selected by Quiznos to provide a system,for ... is a,national chain with over 5000 franchise restaurants, ...
... Alimera Sciences,and pSivida Ltd (Nasdaq: PSDV ... have amended their license and collaboration agreement relating ... for,diabetic macular edema (DME), and other Medidur products. ... of Medidur FA from 50 to 80,percent in ...
... Million Distribution Contract from Anhui ... Huayuan Pharmaceutical Co., ... Co., Inc., (OTC Bulletin Board: TYNP), a manufacturer and,supplier ... today announced that the Company has been granted SFDA,approval ...
Cached Biology Technology:Quiznos Selects Halogen for Fresh Approach to Talent Management 2Alimera Sciences, pSivida Limited Amend Medidur(TM) FA Collaboration Agreement 2Alimera Sciences, pSivida Limited Amend Medidur(TM) FA Collaboration Agreement 3Alimera Sciences, pSivida Limited Amend Medidur(TM) FA Collaboration Agreement 4Tianyin Pharmaceutical Announces SFDA Approval to Market Azithromycin Dispersible Tablets 2Tianyin Pharmaceutical Announces SFDA Approval to Market Azithromycin Dispersible Tablets 3
BD IMag Anti-Mouse CD90.2 (Thy1.2) Particles-DM 2 x 109 cells...
Rabbit polyclonal to Staphylococcus Enterotoxin E (Biotin) ( Abpromise for all tested applications)....
... Ribonuclease III (RNase III) from E. ... double-stranded RNA (dsRNA) to dsRNA fragments that ... dsRNA results in dsRNA fragments of 12-15 ... RNA composed of canonical ribonucleotides as well ...
...
Biology Products: