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:1/22/2015)... York, New York (PRWEB) January 22, 2015 ... filed after Fresenius Medical Care recalled its GranuFlo and ... federal multidistrict litigation underway in U.S. District Court, District ... Order dated January 20th, the Court has remanded a ...
(Date:1/22/2015)... January 22, 2015 Joan Lunden, award-winning ... speaker at the upcoming 32nd Annual Miami Breast ... Physicians’ Education Resource®, LLC (PER®) , shares that “a ... gone undetected if she had not followed up her ...
(Date:1/22/2015)... January 22, 2015 IsoComforter, Inc. has introduced ... with patented IsoTube design to treat specific body parts and ... most progressive and easy to use patented cold therapy machine ... sports related orthopedic and muscle injuries. IsoComforter has become ...
(Date:1/22/2015)... 2015 As interest in the value of ... momentum worldwide, Rev. Eric J. Hall , president and ... (HCCN), will be the keynote speaker on January 27 at ... health system. , The conference, “Hope and Resilience: Innovative and ...
(Date:1/22/2015)... (PRWEB) January 22, 2015 Step into ... at the Science Museum of Virginia. Wicked Plants, the ... Wicked Plants unearths 75 poisonous, carnivorous and diabolical plants ... each room to uncover the biochemical, physical and neurological ...
Breaking Medicine News(10 mins):Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 2Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 3Health News:Court Overseeing Federal GranuFlo Lawsuits Remands Louisiana Claim Back to State Court, Bernstein Liebhard LLP Reports 4Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 2Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 3Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 4Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 5Health News:Joan Lunden Reveals Diagnostic Approach That Led To Early Detection;To Keynote 32nd Annual Miami Breast Cancer Conference® 6Health News:IsoComforter Introduces Best of Class Cold Therapy Product to the Worldwide Market 2Health News:HealthCare Chaplaincy CEO to Keynote Spiritual Care Summit in Israel 2Health News:HealthCare Chaplaincy CEO to Keynote Spiritual Care Summit in Israel 3Health News:Wicked Plants are taking over the Science Museum of Virginia 2
... Campaign for Cancer Prevention (CCP) began in,2007 with the ... care about preventing cancer. It has now,grown to a ... initiative on the social media,platform, Causes.com. Nearly five years ... Women,s,Hospital (BWH), a Harvard Medical School flagship teaching hospital, ...
... male breast cancer is low and most men do not even ... new research from the University of Leeds. Breast cancer ... diagnoses in women in the UK each year. However around 340 ... cancer each year and around 70 men will die. Funded ...
... WASHINGTON, Oct. 3In a feat of technology tweaking that ... University of California, Davis has transformed everyday iPhones into ... cost about as much as a typical app, the ... to perform detailed microscopy and spectroscopy. The team ...
... the University of Bristol, has found that children who are ... more filling. This finding, published (online ahead of print) in ... because it reveals one way in which children over-consume snack ... are at risk of obesity due to consumption of energy-rich ...
... HealthDay Reporter , FRIDAY, Sept. 30 (HealthDay News) ... effective as psychiatric drugs in reducing the agitation that ... suggests. In a three-month period, nursing home residents ... involving music, mime and humorous props showed a significant ...
... accepted for publication in The Endocrine Society,s Journal ... shift work at a young age is associated ... Previous studies have shown that long-term elevated cortisol ... and cardiovascular risk. Shift work, defined as ...
Cached Medicine News:Health News:Campaign for Cancer Prevention connects 6 million+ members on Facebook Causes to research at BWH 2Health News:Campaign for Cancer Prevention connects 6 million+ members on Facebook Causes to research at BWH 3Health News:Researchers call for more awareness of male breast cancer as cases rise 2Health News:Researchers call for more awareness of male breast cancer as cases rise 3Health News:Researchers transform iPhone into high-quality medical imaging device 2Health News:Researchers transform iPhone into high-quality medical imaging device 3Health News:Researchers transform iPhone into high-quality medical imaging device 4Health News:New study finds familiarity increases the fullness that children expect from snack foods 2Health News:Laughter Might Be Good Medicine for Alzheimer's Patients 2Health News:Laughter Might Be Good Medicine for Alzheimer's Patients 3
(Date:1/23/2015)... MERIDIAN, Idaho , Jan. 23, 2015  Now available ... Patent No. 7,849,611) support that prevents ankle sprains by ... a brace, it fits securely around the outside of ... ankle mobility & comfort while still offering protection against ...
(Date:1/23/2015)... Cord Blood America, Inc. ( www.cordblood-america.com ) (OTC Bulletin Board: ... Preliminary Proxy Statement, Schedule 14A, with the U.S. Securities & ... for shareholders detailing why a "YES" vote to increase the ... As we reflect upon the 2014 fiscal year ...
(Date:1/23/2015)... , Jan. 23, 2015  Mallinckrodt plc (NYSE: ... has approved a share repurchase program. The open-ended authorization ... of Mallinckrodt plc ordinary shares. "Funding ... top priorities as we continue to pursue a range ...
Breaking Medicine Technology:The Armor1 Ankle Roll Guard is the New Breakthrough Solution for Ankle Sprain Prevention! 2Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 2Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 3Cord Blood America Announces Preliminary Proxy Filing and Details Why a "YES" Vote to Increase Authorized Shares Is Requested 4Mallinckrodt plc Announces Share Repurchase Program 2Mallinckrodt plc Announces Share Repurchase Program 3Mallinckrodt plc Announces Share Repurchase Program 4
... Fla., Nov. 18 /PRNewswire-FirstCall/ -- AstraZeneca (NYSE: ... phase II studies, ONSET/OFFSET and RESPOND for ticagrelor (BRILINTA(TM)) ... in Orlando, FL,(1,2) with ONSET/OFFSET study results being simultaneously ... The ONSET/OFFSET data showed that treatment with ...
... , MADISON, N.J., Nov. 18 Quest Diagnostics Incorporated (NYSE: ... and services, today announced that the Simplexa Influenza A H1N1 (2009) ... and is now available as a test kit for distribution to ... at MEDICA 2009, November 18 to 21, 2009, at the Dusseldorf ...
Cached Medicine Technology:Results From Two Phase II Trials Add to Understanding of Ticagrelor (BRILINTA(TM)) and How it Works in the Body 2Results From Two Phase II Trials Add to Understanding of Ticagrelor (BRILINTA(TM)) and How it Works in the Body 3Results From Two Phase II Trials Add to Understanding of Ticagrelor (BRILINTA(TM)) and How it Works in the Body 42009 H1N1 Flu Test From Quest Diagnostics Now CE Marked For International Distribution Under The Simplexa(TM) Brand Name 22009 H1N1 Flu Test From Quest Diagnostics Now CE Marked For International Distribution Under The Simplexa(TM) Brand Name 32009 H1N1 Flu Test From Quest Diagnostics Now CE Marked For International Distribution Under The Simplexa(TM) Brand Name 42009 H1N1 Flu Test From Quest Diagnostics Now CE Marked For International Distribution Under The Simplexa(TM) Brand Name 5
... Anatomic Knee system was designed to ... all articulating surfaces, to minimize contact ... achieve a well balanced knee with ... The MJS Knee system allows maximum ...
... of digital mobile imaging just got ... software and instrumentation. Now using,your C-arm ... Easily Add Visualization and Navigation ... Accelerate Intraoperative Decision Making, Provide ...
... GE's surgical navigation for ... system to integrate C-arm ... technology. It features automatic ... image to the patient's ...
... on over 20 years of CT engineering ... is at the forefront of the revolution ... acquire volumetric data at high speed with ... based on extensive clinical experience and close ...
Medicine Products: