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:8/28/2015)... MO (PRWEB) , ... August 28, 2015 , ... ... testosterone lawsuits now consolidated as MDL 2545, Testosterone Replacement Therapy Products ... a cap on the number of custodial files the PSC may receive for ...
(Date:8/28/2015)... ... ... Super-Sod launched their new website this summer. The new user-friendly ... the responsive web design. Website users will now have an improved browsing experience on ... sod, sign-up for the monthly turf-tips email, and find a local Super-Sod store all ...
(Date:8/28/2015)... ... 2015 , ... The Wilson and Juanita Mann Health Center ... a patient’s quality of life through early diagnosis and treatment. The focus is ... HIV/AIDS, STDs, and others. A Women’s, Infants & Children (WIC) clinic also provides ...
(Date:8/27/2015)... ... August 28, 2015 , ... Grace Bay Beach has been recognized ... to an already impressive list of accolades for the Turks and Caicos so far ... beach in the world by TripAdvisor, the world's leading authority on travel. , ...
(Date:8/27/2015)... ... ... Mesa Community College graduate, Andrew Sypher (Mesa), is one of 10 ... and engagement, to receive a Phi Theta Kappa 2015 Hites Transfer Scholarship, to assist ... a $7,500 scholarship to assist in his goal of becoming a foreign diplomat with ...
Breaking Medicine News(10 mins):Health News:National Testosterone Lawyers Provide Testosterone Lawsuit MDL Update 2Health News:National Testosterone Lawyers Provide Testosterone Lawsuit MDL Update 3Health News:National Testosterone Lawyers Provide Testosterone Lawsuit MDL Update 4Health News:Health Clinic Opening and Public Art Unveiling 2Health News:Grace Bay Beach Acknowledged as a Top Caribbean Beach for Weddings 2Health News:Mesa Community College Graduate Andrew Sypher Receives Hites Transfer Scholarship 2Health News:Mesa Community College Graduate Andrew Sypher Receives Hites Transfer Scholarship 3
... College Park, MD (November 30, 2007) Concerns over possible ... in the New England Journal of Medicine should not scare ... scans play a critical role in saving the lives of ... the American Association of Physicists in Medicine (AAPM). , In ...
... of relationships between parents and their adult children improve ... researcher has found. , Karen Fingerman, an associate professor ... Consumer and Family Sciences, examined relationships adults 70 and ... offspring. The parents in the study also suffered either ...
... a third, study found , , FRIDAY, Nov. 30 (HealthDay ... significantly slows the decline in lung function seen in ... team at Case Western Reserve University School of Medicine, ... took high doses of ibuprofen twice a day had ...
... and Ceramic Oxides, BATON ROUGE, La., Nov. ... world leader in the development and marketing,of aluminum ... and other industrial applications, will increase the prices ... percent, effective globally on all,shipment made on or ...
... Ltd., (Amex: CPD ) announced today that ... tentative approval for the Company,s,Abbreviated New Drug Application ... mg, and 20 mg., Escitalopram is indicated ... approval is for the generic bioequivalent product,to Lexapro(R), ...
... Enhance Cardiovascular Fitness, Improve Health and Balance, and Promote ... ... today unveiled a revolutionary new fitness system that combines the,benefits of dance ... the first time to the adult market., This innovative and groundbreaking ...
Cached Medicine News:Health News:Hazards of CT scans overstated 2Health News:Aging improves parent, child relationships, research shows 2Health News:Ibuprofen Preserves Lung Function in Kids With Cystic Fibrosis 2Health News:Ibuprofen Preserves Lung Function in Kids With Cystic Fibrosis 3Health News:Albemarle Raises Prices of Specialty Aluminas 2Health News:Caraco Pharmaceutical Laboratories, Ltd. Announces Tentative FDA Approval for Generic Lexapro(R) 2Health News:The Dancetown Fitness System: a Radical New Approach to Physical Exercise for Adults Combines State-of-the-Art Computer Technology and Dance 2
(Date:8/27/2015)... 27, 2015 According to a new market ... Seating, Universal, Standing, Bariatric ), Material (Nylon, Padded, Canvas), Usage Type ... published by MarketsandMarkets, the Medical Lifting Sling Market is poised to ... 2015, at a CAGR of 11.4 % from 2015 to 2020. ... rket data T ables and 65 ...
(Date:8/27/2015)... Pa. , Aug. 27, 2015  Now that ... the Da Vinci S™ surgical robot, hospitals with robotic ... plans. They also need to see where they stand ... on the right patients? Have they adopted best practices ... surgery program in the future? ECRI Institute, ...
(Date:8/27/2015)... 2015   Proxsys Rx, the nation,s ... expanding its population health management services with ... care program for hospitals and health systems. ... with technology systems and pharmacy support services ... been mandated by Center for Medicare and ...
Breaking Medicine Technology:Medical Lifting Sling Market Worth USD 706.0 Million by 2020 2Medical Lifting Sling Market Worth USD 706.0 Million by 2020 3Medical Lifting Sling Market Worth USD 706.0 Million by 2020 4ECRI Institute's New Robotic Surgery Service Helps Hospitals Brace for Change 2ECRI Institute's New Robotic Surgery Service Helps Hospitals Brace for Change 3Rx Care Management by Proxsys Rx - Transition of Care Technology Helps Prevent Hospital Medication Errors 2
... AirPal ®, a leading innovator and supplier ... the expansion of its line of air-activated transfer pads ... announcement marks the launch of an entire new series ... President AirPal, Inc.  "Among other functions, these exciting new ...
... recently merged with World Wide Relics (OTC Bulletin Board: ... its name to China PharmaHub Corp, is proud to ... Yongkon Pharmacy Co. Ltd., ("Yongkon"), a pharmaceutical company based ... 2600 hospitals in China.  PharmaHub was authorized by Yongkon ...
Cached Medicine Technology:AirPal Expands Line of Air-Activated Transfer Pads to Include Multi-Purpose Patient Positioning Functionality 2China PharmaHub Signs Agreement with Chengdu Yongkon Pharmacy 2China PharmaHub Signs Agreement with Chengdu Yongkon Pharmacy 3
... a highly featured laser doppler system ... perfusion from multiple tissue sites. This ... Digital Signal Processing (DSP) and laser ... yet simple to use, multi-channel monitor, ...
Transcranial vascular DWL doppler....
Transcranial vascular DWL doppler....
... product from Viasys Neurocare is a full-featured physiologic ... portable size. It contains many of the features ... offered at an economical price. , ,The smaller ... a crowded lab environment, the physician's office, a ...
Medicine Products: