Navigation Links
Gene therapy shows promise as hemophilia treatment in animal studies
Date:11/2/2011

WINSTON-SALEM, N.C. -- For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

Researchers at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine, collaborating with other institutions, report in Experimental Hematology that a single injection of genetically-modified adult stem cells in two sheep converted the severe disorder to a milder form. The journal is a publication of the Society for Hematology and Stem Cells

"A new approach to treating severe hemophilia is desperately needed," said lead author Christopher D. Porada, Ph.D., associate professor of regenerative medicine at Wake Forest Baptist. "About 75 percent of the world doesn't have access to the current treatment therapy to replace missing clotting factors. This puts patients in most of the world at risk of severe and permanent disabilities."

Porada cautioned that challenges will need to be overcome before the treatment can be applied to humans, including that the sheep developed an immune response to the therapy that could decrease its effectiveness and duration.

There is currently no cure for the rare bleeding disorder hemophilia. People with this genetic disorder lack a protein, known as a clotting factor, needed for normal blood clotting. As a result, they may bleed for a longer time than others after an injury, as well as bleed internally, especially in joints such as the knees, ankles, and elbows. This bleeding can damage the organs and tissues and be life threatening. Even when life-threatening bleeds are prevented with replacement therapy, it doesn't prevent smaller bleeds within the joints that can cause pain and decreased mobility.

People with hemophilia A, the most common type, are missing clotting factor VIII. For the study, the researchers used a combined stem cell/gene therapy approach to increase levels of factor VIII produced by the animals.

The scientists first inserted a gene for factor VIII into engineered mesenchymal stem cells, a type of adult stem cell. The cells acting as a carrier for the gene were then injected into the abdominal cavity of the sheep. The scientists selected mesenchymal stem cells to carry the gene because they have the ability to migrate to sites of injury or inflammation.

In the treated animals, the cells migrated to the joints and stopped ongoing bleeding. In addition, all spontaneous bleeding events ceased, and the existing joint damage was completely reversed, restoring normal posture and gait to these crippled animals, and enabling them to resume a normal activity level.

However, a paradox of the treatment was that while the symptoms were eliminated, the sheep developed an immune response to factor VIII, suggesting that the treatment's effects would be reduced or shorter in duration. The scientists are currently working to learn why the immune response occurred and to develop strategies to prevent it.

"While preliminary, these findings could pave the way for a new therapy for hemophilia patients who experience debilitating bleeding in their joints," Porada said.


'/>"/>
Contact: Karen Richardson
krchrdsn@wakehealth.edu
336-716-4453
Wake Forest Baptist Medical Center
Source:Eurekalert

Related medicine news :

1. Nicotine replacement therapy is over-promoted since most ex-smokers quit unassisted
2. Social Anxiety and Panic - Alternative Treatment to Drugs and Therapy
3. Longview Therapy Center, PLLC, Unveils Its Definition Of Hope
4. Short-term radiation therapy successful on breast cancer
5. ASCROs Response to NY Times Articles on Radiation Therapy Incidents
6. Children With Cerebral Palsy Benefitting From New Physical Therapy Regimen
7. DavisPTnetwork Partners with the New York Physical Therapy Association to Provide Online Continuing Education
8. Behavioral therapy improves sleep and lives of patients with pain
9. WHI data confirm short-term heart disease risks of combination menopausal hormone therapy
10. Promising therapy for relapsing multibple sclerosis
11. Split-course palliative radiotherapy confirmed as effective treatment for advanced NSCLC
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/26/2017)... ... 26, 2017 , ... Amir Qureshi, MD is the first physician in Arkansas ... stimulation system. The Nuvectra™ Algovita SCS System has been FDA approved as a treatment ... in Arkansas to introduce the most powerful SCS system and the only stretchable lead ...
(Date:5/26/2017)... ... May 26, 2017 , ... On May 24, the ... narrowly passed the U.S. House on May 4, would result in 23 million Americans ... continued implementation of the 2010 Patient Protection and Affordable Care Act (ACA). , ...
(Date:5/26/2017)... ... May 26, 2017 , ... Dr. Alex Rabinovich, a ... announce a new, informational blog post on insurance options. If a Bay Area patient ... may help save time and money. Visiting an in-network provider for a second opinion ...
(Date:5/26/2017)... , ... May 26, 2017 , ... “Cactus Jack: Against ... on so many others. “Cactus Jack: Against All Odds” is the creation of ... Walter D. Hubbard is married to Jack Carlisle’s third child Jane. Walter. Walter ...
(Date:5/24/2017)... Hills, CA (PRWEB) , ... May 24, 2017 , ... ... announced today its participation in nVerge 2017 – a one-day technology conference in San ... Altec’s document management solution, which allows users to fully utilize and enhance their Sage ...
Breaking Medicine News(10 mins):
(Date:5/10/2017)... 10, 2017  The Corporate Whistleblower Center says, ... sleep therapy clinics to call us anytime at ... is involved in a substantial scheme to overbill ... from an employee of a medical equipment company ... to provide medical practice groups with extra generous incentives ...
(Date:5/9/2017)... , May 9, 2017  Oramed Pharmaceuticals ... ), a clinical-stage pharmaceutical company focused on the ... that the Canadian Intellectual Property Office has granted ... Oral Administration of Exenatide". The patent covers Oramed,s ... GLP-1 is an incretin hormone that ...
(Date:5/6/2017)... , May 5, 2017  May is Stroke ... one of the most important methods to prevent a ... the Centers for Disease Control and Prevention, undetected and ... 1 Omron, the global leader in personal heart ... the elimination of heart attack and stroke and is ...
Breaking Medicine Technology: