Navigation Links
Researchers improve gene therapy technique for children with immune disorder
Date:9/11/2012

By including chemotherapy as a conditioning regimen prior to treatment, researchers have developed a refined gene therapy approach that safely and effectively restores the immune system of children with a form of severe combined immunodeficiency (SCID), according to a study published online today in Blood, the Journal of the American Society of Hematology (ASH).

SCID is a group of rare and debilitating genetic disorders that affect the normal development of the immune system in newborns. Infants with SCID are prone to serious, life-threatening infections within the first few months of life and require extensive treatment for survival beyond infancy.

Adenosine deaminase (ADA) deficiency, which accounts for approximately 15 percent of all SCID cases, develops when a gene mutation prohibits the production of ADA, an enzyme that breaks down toxic molecules that can accumulate to harmful levels and kill lymphocytes, the specialized white blood cells that help make up the immune system. In its absence, infants with ADA-deficient SCID lack almost all immune defenses and their condition is almost always fatal within two years if left untreated. Standard treatment for ADA-deficient SCID is a hematopoietic stem cell transplant (HSCT) from a sibling or related donor; however, finding a matched donor can be difficult and transplants can carry significant risks. An alternate treatment method, enzyme replacement therapy (ERT), involves regular injections of the ADA enzyme to maintain the immune system and can help restore immune function; however, the treatments are extremely expensive and painful for the young patients and the eff
'/>"/>

Contact: Claire Gwayi-Chore
cgwayi-chore@hematology.org
202-776-0544
American Society of Hematology
Source:Eurekalert

Page: 1 2 3 4

Related medicine news :

1. Moffitt researchers say race affect likelihood of finding suitable stem cell donor
2. Researchers closer to understanding actions of cells involved in atherosclerosis
3. Case Western Reserve researchers create short-term memories in-vitro
4. Researchers find first evidence for a genetic cause for Barretts esophagus
5. Researchers show cost-effectiveness of HIV testing in drug abuse treatment programs
6. When clinicians and researchers look outside the box
7. Moffitt Cancer Center researchers study childhood melanoma characteristics
8. Pint-size microRNAs show promise against weighty problem, researchers say
9. University of Hawaii cancer researchers discover gene defect responsible for cancer syndrome
10. Moffitt Cancer Center researchers find acidic pH microenvironments in tumors aid tumor cell survival
11. BUSM/VA researchers examine new PTSD diagnosis criteria
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/22/2014)... 22, 2014 The Louis ... is dedicated to distribute health information technology innovation ... worldwide announced the development of six key guiding ... when receiving healthcare. These are an expansion of ... “providing care that is respectful of and responsive ...
(Date:10/22/2014)... 22, 2014 With the Affordable Care Act ... by the American Institutes for Research (AIR) finds that three ... use health insurance, but 42 percent say they are not ... before signing up for coverage. , The AIR survey found ... percent able to calculate correctly how much they owe for ...
(Date:10/22/2014)... Cheap Hosting USA is among the most distinguished review ... that iPower ( http://www.best-cheap-hosting-usa.com/go/iPower/ ) is the best cheap ... , “iPower is one of the most reliable hosting ... of useful products for new and old clients. What’s ... and a number of features which will appeal to ...
(Date:10/22/2014)... Your Body’s Way , In a world where a multi-billion-dollar ... can be difficult to choose the right weight loss method. ... it the healthy way? Set aside the fads and resist ... regularly, and choose foods with the most nutritional benefits. ... exercise more and eat less. Despite the number of advertisements ...
(Date:10/22/2014)... Lintelus, Inc., an event software provider, announced ... will be speaking on the subject of “Event Technology ... 28, at the Holiday Inn in New York City. ... as a Tech Demo at 12:45pm, to educate event ... engaging event experience. , The Annual Meetings Technology Expo ...
Breaking Medicine News(10 mins):Health News:The Sullivan Institute for Healthcare Innovation Announces Release of Guiding Principles for Patient Experience-Centered Care 2Health News:Many Americans Fail to Ask Basic Questions Before Signing Up for Health Insurance, National Survey Finds 2Health News:Many Americans Fail to Ask Basic Questions Before Signing Up for Health Insurance, National Survey Finds 3Health News:Many Americans Fail to Ask Basic Questions Before Signing Up for Health Insurance, National Survey Finds 4Health News:Best Cheap Hosting USA Has Recently Announced The Best Cheap Web Hosting Companies In 2014 2Health News:ReductionGuruMD Presents Ways to Improve Your Weight Loss Results 2Health News:Jay Cormier, Sr. Vice President of Sales for Lintelus, Speaks about Event Technology Integration at Meetings Technology Expo in NYC 2
... Cancer Center is teaming up with ENT and Allergy ... between Mount Sinai and ENT and Allergy Associates, the ... in the tri-state area, together will provide patients with ... and advanced surgical options for patients diagnosed with cancer ...
... 12 A new, free white paper,details how ... has,substantially reduced lift injuries while boosting staff satisfaction ... that provides patient transfer,and mobility assistance. Available ... is provided by the Safe Lifting Portal, (http://www.safeliftingportal.com ...
... Integrated, Inc., a,pioneer in health and condition (disease) ... ground-breaking Synergy Personal Health,Management program at DMAA,s Disease ... 16th at Caesar,s Palace in Las,Vegas. With ... need to stay,informed of the best practices, latest ...
... treatment for breast cancer, dramatically reduces symptoms of the ... standard medications for the mental illness, a new study ... National Institute of Mental Health (NIMH) who conducted the ... called protein kinase C (PKC) that regulates activities in ...
... Routine helps teens make better food choices as adults, study ... eat dinner with their families on a regular basis are ... adults, a new study shows. , These teens also go ... found. , Reporting in the Journal of the American ...
... NASHUA, N.H., Sept. 12 iCAD, Inc. (Nasdaq:,ICAD), ... the early identification of cancer, today announced that ... will be presenting at,the America,s Growth Capital 4th ... 12:00 p.m. (Eastern Daylight Time) at the,Sheraton Boston. ...
Cached Medicine News:Health News:Mount Sinai & ENT and Allergy Associates, LLP affiliate to fight cancer 2Health News:New Case Study Details How an Illinois Hospital Created a Safe Lifting Team that Dramatically Reduced Injuries, Increased Staff Satisfaction 2Health News:Health Integrated to Showcase Synergy Personal Health Management(R) at DMAA Conference 2Health News:Manic phase of bipolar disorder benefits from breast cancer medication 2Health News:Manic phase of bipolar disorder benefits from breast cancer medication 3Health News:iCAD to Present at America's Growth Capital 4th Annual Emerging Growth Conference 2
(Date:10/22/2014)... -- Investor-Edge has initiated coverage on the ... ), Progenics Pharmaceuticals Inc. (NASDAQ: PGNX ), ... Pharmaceuticals Inc. (NASDAQ: IDRA ), and Merrimack ... companies can be accessed at: http://investor-edge.com/register . ... ended at 4,419.48, up 2.40%, the Dow Jones Industrial ...
(Date:10/22/2014)... RIDGE, N.J. , Oct. 22, 2014 /PRNewswire/ ... (RMANJ), a world-renowned leader in the field of ... rapid approach to embryonic screening. The research abstract, ... Society for Reproductive Medicine (ASRM) meeting in ... screening (CCS) and single gene defect (SGD) pre-implantation ...
(Date:10/22/2014)... AVACEN Medical announced today that its AVACEN ... clearance by the U.S. Food and Drug Administration. This ... the Internet for: the temporary relief of joint ... and sprains . Logo - http://photos.prnewswire.com/prnh/20141021/153513 ... is a sophisticated medical device incorporating over 3,000 firmware ...
Breaking Medicine Technology:Market Momentum on Biotech Stocks - Amicus Therapeutics, Progenics Pharma, Threshold Pharma, Idera Pharma, and Merrimack Pharma 2Market Momentum on Biotech Stocks - Amicus Therapeutics, Progenics Pharma, Threshold Pharma, Idera Pharma, and Merrimack Pharma 3Market Momentum on Biotech Stocks - Amicus Therapeutics, Progenics Pharma, Threshold Pharma, Idera Pharma, and Merrimack Pharma 4Market Momentum on Biotech Stocks - Amicus Therapeutics, Progenics Pharma, Threshold Pharma, Idera Pharma, and Merrimack Pharma 5New IVF Research Highlights Improved Genetic Screening Approach To Help Parents Have Healthier Babies 2New IVF Research Highlights Improved Genetic Screening Approach To Help Parents Have Healthier Babies 3AVACEN Medical Announces FDA Clearance of First Medical Device with Internal Game Mode 2
... Presented in Spine, Lung, Liver and Prostate Treatment Emphasized the Benefits of ... , ... ARAY ), a global leader in the field of radiosurgery, today ... Radiotherapy Workshop, held in Brussels, Belgium on May 28 ...
... July 6 Questcor Pharmaceuticals, Inc. (NASDAQ:  QCOR) announced today ... Board of Directors. Dr. Blutt, 53, is the Founder and Chief Executive ... that is a shareholder of Questcor. Dr. Blutt is also board-certified in ... Medical College. , , , ...
Cached Medicine Technology:Presentations at the Third European Stereotactic Radiation Therapy and Whole Body Radiotherapy Workshop Highlight Clinical Outcomes and Emerging Trends 2Presentations at the Third European Stereotactic Radiation Therapy and Whole Body Radiotherapy Workshop Highlight Clinical Outcomes and Emerging Trends 3Presentations at the Third European Stereotactic Radiation Therapy and Whole Body Radiotherapy Workshop Highlight Clinical Outcomes and Emerging Trends 4Presentations at the Third European Stereotactic Radiation Therapy and Whole Body Radiotherapy Workshop Highlight Clinical Outcomes and Emerging Trends 5Mitchell J. Blutt, M.D. Appointed to Questcor Board of Directors 2Mitchell J. Blutt, M.D. Appointed to Questcor Board of Directors 3Mitchell J. Blutt, M.D. Appointed to Questcor Board of Directors 4Mitchell J. Blutt, M.D. Appointed to Questcor Board of Directors 5
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
Medicine Products: