Navigation Links
Gene Rx May Fight Severe Blood Disorder
Date:9/15/2010

e trials are sponsored by the Cambridge, Mass.-based bluebird bio.

The report is published in the Sept. 16 issue of Nature.

Based on their initial success with gene therapy, Leboulch's group plans to treat another two patients. If they also respond well, they will enroll "a larger cohort" of patients, he said.

In addition, the researchers plan to treat patients who have sickle cell disease with the gene therapy as well, Leboulch said. The same technique can be used for both diseases, he said.

Leboulch concedes that the treatment is very expensive. But he hopes that if it becomes widely used, the price will drop, eventually costing less than a lifetime of monthly transfusions and chelating drugs.

Dr. Mustafa Tekin, an associate professor at the John P. Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine, said that "gene therapy has been long awaited for thalassemia."

"This is the first example of a successful gene therapy for beta-thalassemia," he said. "This brings great hope for patients for the final cure for the disorder."

However, there are possibilities of adverse effects, Tekin said. He joined the researchers in cautioning that treatment to date has been limited to just one patient.

Tekin said that the gene that absorbed the therapeutic gene is associated with certain cancers, such as leukemia. While the patient does not have signs of the cancer, it is important "to follow this patient for the long-term for signs of leukemia," he said.

More trials and patients are needed to really assess the effects and side effects of the treatment, Tekin said. Whether it will treat other forms of thalassemia isn't known, he added.

"One patient is important and a significant achievement, but you still need to see more patients to make sure that it works for many patients and also that it works safely," he said.

More information

For more informati
'/>"/>

Copyright©2010 ScoutNews,LLC.
All rights reserved  

Page: 1 2 3 4

Related medicine news :

1. UCLA selected by Homeland Security to help establish guidelines for firefighter health and safety
2. Addressing negative thoughts most effective in fighting loneliness
3. In Early Trial, Targeted Therapy Fights Advanced Melanoma
4. UM School of Medicine finds mentorship program successfully fights childhood obesity
5. Breaching the blood-brain barrier to create powerful new tools for fighting cancer
6. UCLA scientists create army of tumor-fighting immune cells and watch as they attack cancer
7. UCI to get $4.7 million to fight malaria in Southeast Asia
8. The Kristen Renee Foundation Continues to Fight for Frederick, MD for Answers from the EPA and Fort Deterick
9. The Quantum Scale Helps in the Fight Against Childhood Obesity
10. Fighting Off the Freshman Fifteen
11. Molecular signatures may aid fight against pediatric liver disease
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Gene Rx May Fight Severe Blood Disorder 
(Date:8/30/2014)... (PRWEB) August 30, 2014 Serving contented ... in India, Profit By Outsourcing is now offering ... services. The company gives emphasize on putting forward the ... product to clients. Their dedicated team of professionals and ... results. They can handle even the critical issues of ...
(Date:8/30/2014)... Saturday 30 August 2014: Transvenous lead extraction (TLE) is ... to data from the European Lead Extraction ConTRolled (ELECTRa) ... Congress 2014 by Dr Maria Grazia Bongiorni, chair of ... number of cardiac implantable electronic devices (CIED) implant procedures ... the number of complications has increased and leads are ...
(Date:8/30/2014)... Top10BestSEOHosting.com is a well-known review website. Recently, the ... http://secure.hostgator.com/~affiliat/cgi-bin/affiliates/clickthru.cgi?id=seohosts is the best Windows Hosting supplier ... that Bluehost and Arvixe are excellent companies for ... Top 10 Web Hosting in 2014, is a ... offers rich features and great Windows hosting solutions ...
(Date:8/30/2014)... OK (PRWEB) August 30, 2014 LiveStreamingFitness.com, a ... well as other healthy lifestyle resources, is proud to announce ... nationwide resources for at home fitness solutions by ABC’s Good ... recent featured story on ABC’s Good Morning America, “Live Streaming ... you can streamline your day by streamlining your workout…to boot ...
(Date:8/30/2014)... McLeod's Sunset Park Auto Service , a family ... ties to the community celebrates sixty years in business. In ... been chosen by The Tampa Tribune’s readers again ... Tim McLeod’s father, Norris (most know him as Mac) opened ... son, Tim runs the day to day, taking on the ...
Breaking Medicine News(10 mins):Health News:Profit By Outsourcing Offering Discount for Labor Day on all Web Development Services 2Health News:Transvenous lead extraction clinically successful in 98 percent of cases 2Health News:Transvenous lead extraction clinically successful in 98 percent of cases 3Health News:Top10BestSEOHosting.com: HostGator.com Is The Best Windows Hosting Supplier 2Health News:LiveStreamingFitness.com is New Trend In Fitness 2Health News:McLeod's Sunset Park Auto Service Celebrates 60 Years Honored with Best of South Tampa 2
... 3 Inverness Medical,Innovations (Amex: IMA ) today ... of both core laboratory and point of care,diagnostic testing ... addition Inverness will pay an earn out up to ... Bio-Stat had,revenues of approximately $29.5m in 2006 and has ...
... Group wants to buy 27 nursing homes in ... DETROIT, Oct. 3 SEIU Healthcare Michigan launched a,radio ... Wall,Street investment firm,s planned purchase of 27 nursing homes in ... the homes, endanger seniors,and send nurses to the unemployment line ...
... study brings relief to new parents who, while navigating a ... through as many as 15 pokes by his or her ... Journal of Pediatrics, shows that no efficacy or safety is ... the process in effect, tripling up three of the ...
... ,Unilens Vision Inc. (OTC Bulletin Board: UVICF) (TSX Venture ... has been named,the exclusive worldwide distributor of the UniVision(R) ... as the exclusive distributor,for North and South America and ... in the rest of the world. "We feel ...
... and Services for AARP,s 39 Million,Members, ... new catalog, "AARP Health Essentials by Walgreens" ... provide AARP,s 39 million members,access to discounted ... catalog,and website offers a 5 percent discount, ...
... (Amex: PTG ), a leading supplier of ... center solutions,announced today that its SI Systems, brand ... an automated light-picking system and related,software modules for ... The contract calls for SI Systems to provide ...
Cached Medicine News:Health News:Inverness Medical Innovations Acquires Bio-Stat Healthcare Group 2Health News:SEIU Launches Ad Campaign Opposing Corporate Raider's Plan to Buy Mich. Nursing Homes 2Health News:Combination vaccines okay for infants, study shows 2Health News:Combination vaccines okay for infants, study shows 3Health News:Unilens Names Eschenbach Optik Exclusive Worldwide Distributor of the UniVision(R) Low Vision Lens 2Health News:Unilens Names Eschenbach Optik Exclusive Worldwide Distributor of the UniVision(R) Low Vision Lens 3Health News:Walgreens Launches New Health Essentials Catalog and Website for AARP Members Featuring More Than 20,000 Health-Related Products 2Health News:Walgreens Launches New Health Essentials Catalog and Website for AARP Members Featuring More Than 20,000 Health-Related Products 3Health News:Paragon Technologies' SI Systems Brand Awarded A $1.0 Million System Expansion Contract for an Automated Light-Picking System 2Health News:Paragon Technologies' SI Systems Brand Awarded A $1.0 Million System Expansion Contract for an Automated Light-Picking System 3
(Date:8/29/2014)... , August 29, 2014 ... Seed Oil Market by Extraction type (SFE, Cold ... & Personal Care, Pharmaceutical, Food Supplements, Fragrance, & Others) ... by MarketsandMarkets, defines and segments the Amaranth Seed ... the market size in terms of value. The ...
(Date:8/29/2014)... Corporation , a neurodiagnostics medical device company based in ... Forcht Dagi , MD, DmedSc, MPH, MBA, FACS, FRCSEd  was ... " Teo Forcht Dagi is an experienced leader with ... said Remis Bistras , President and CEO of Vittamed. ... entrepreneurial expertise to our Board." Dr. Dagi is ...
(Date:8/29/2014)... , Aug. 29, 2014 /PRNewswire-iReach/ -- ChemImage will ... the Australian and New Zealand Forensic Science Society,s ... Sciences in Adelaide, Australia . ... concerning the advancements that hyperspectral imaging can bring ... Jeffrey Beckstead , Director of Product Development at ...
Breaking Medicine Technology:Amaranth Seed Oil Market worth $700.6 Million by 2019 2Amaranth Seed Oil Market worth $700.6 Million by 2019 3Amaranth Seed Oil Market worth $700.6 Million by 2019 4Vittamed Adds Prominent Neurosurgeon and Venture Capitalist T. Forcht Dagi to Board of Directors 2ChemImage to Discuss Hyperspectral Imaging for Forensic Investigation at the ANZFSS 22nd International Symposium on the Forensic Sciences 2ChemImage to Discuss Hyperspectral Imaging for Forensic Investigation at the ANZFSS 22nd International Symposium on the Forensic Sciences 3
Femoral selective catheter. Catheter material used is polyurethane (DUCOR). Guidewire campatibility is 0.038". Packaging 5/Box....
Femoral selective diagnostic catheter.The material used is polyurethane (DUCOR). All selective shapes have thrulumen design. Guidewire campatibility is 0.038". Packaging 5/Box....
Cordis DUCOR Catheters are available in a broad variety of French sizes and configurations. These catheters combine an atraumatic tip with either a braided or non-braided body....
... has a complete offering of cardiology catheters. ... these catheters are designed with excellent torque, ... and better push-ability, and large inner lumens ... 5, 6 and 7 French sizes with ...
Medicine Products: