Navigation Links
New approach to sickle-cell disease shows promise in mice
Date:12/7/2009

A new genetic approach to treating sickle cell disease is showing promising results in mice, report researchers from Children's Hospital Boston. By inactivating a gene they previously discovered to be important in the laboratory, they were able to boost production of a healthy fetal form of hemoglobin in the mice, potentially compensating for the defective adult hemoglobin that causes red blood cells to "sickle" and obstruct blood flow.

The study was presented by first author Jian Xu, PhD, on Sunday, December 6, at the American Society for Hematology meeting in New Orleans, at a 3 p.m. Plenary Scientific Session.

Currently, there are only a limited number of therapies available for patients with sickle cell disease, the most common inherited blood disorder in the U.S., says senior study author Stuart H. Orkin, MD, of Children's Division of Hematology/Oncology, also David G. Nathan Professor of Pediatrics at Harvard Medical School.

Shortly after birth, babies switch from producing the fetal form of hemoglobin, the protein inside red blood cells that carries oxygen, to producing the adult form the type that is affected in sickle cell disease. It's long been known that people who retain the ability to produce fetal hemoglobin have much milder disease. In previous studies (http://www.childrenshospital.org/newsroom/Site1339/mainpageS1339P1sublevel485.html), the Children's researchers, with collaborators, found that a gene called BCL11A is involved in switching off fetal hemoglobin production in adults. Working with genetically engineered mice, they then explored whether that switch could be turned back on to alleviate the disease.

In embryonic mice, inactivation of the BCL11A gene led to a robust expression of gamma-globin (the long protein chains making up the fetal form of hemoglobin) during late gestation: more than 90 percent of the globin produced was of this fetal type. In adult mice (8-10 weeks old), inactivation of the BCL11A gene in the blood system resulted in more than a 1,000-fold increase in gamma-globin production in bone marrow erythroblasts (the precursors to red blood cells) as compared with control mice. This increase was rapid and persisted during the course of the experiments (up until the mice were 25 weeks old).

This line of research began with comprehensive gene association studies, published in 2008 with collaborators at the Broad Institute of Harvard and MIT (http://www.childrenshospital.org/newsroom/Site1339/mainpageS1339P1sublevel452.html). These studies, involving 1600 patients with sickle cell disease, identified five DNA sequence variants (altered strings of genetic code) that correlated with fetal hemoglobin levels. BCL11A, on chromosome 2, had the largest effect, and Orkin and Vijay Sankaran, an MD-PhD student working with Orkin, later demonstrated that this gene directly suppresses fetal hemoglobin production.

If these preliminary results in mice hold up in human studies, inactivating BCL11A may also help patients with thalassemia, another blood disorder involving abnormal hemoglobin, adds Orkin.


'/>"/>

Contact: Rob Graham
rob.graham@childrens.harvard.edu
617-919-3110
Children's Hospital Boston
Source:Eurekalert

Related medicine news :

1. Buchang Global Health Forum 2009: An Innovative Approach for Management of H1N1 Swine Flu
2. Brandeis studies evaluate visionary approach to improving eyesight
3. TMJ and Chiropractic Care: An Overall Wellness Approach
4. Endologix Receives IDE Approval for Fully Percutaneous Approach to EVAR
5. Dallas Facial Plastic Surgeon Publishes Cutting-Edge Novel Approach To Facelift Technique
6. Avizents New TeamCOMP Approach Helps Ensure Lower Workers Comp Costs and Faster Claims Resolution
7. Regence Takes Community Approach to Provider Reviews
8. Depression in older cancer patients can be effectively treated with collaborative approach
9. Inventive combination of research approaches identifies new target for treating leukemia
10. New approach for the treatment of malignant brain tumors
11. Global Regulators to Discuss Benefit-Risk Approaches of Medicinal Products at DIA Workshop
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/8/2016)... ... 08, 2016 , ... Brenntag Specialties, Inc.(BSI) , is ... & Beverage and Dietary Supplement market segments across the western ... geographies east of the Rocky Mountains since 2012. Consistent performance in business ...
(Date:2/8/2016)... ... ... The Valentine’s Season is famous for gift giving with flowers, chocolates and other tokens of ... This year, for more than 5.6 million Americans suffering with Alzheimer’s, those store bought gifts ... them of the lives they’ve led and the people they’ve touched. , That’s ...
(Date:2/8/2016)... ... February 08, 2016 , ... If you are feeling ... handle, you are not alone. According to the Center for Disease Control and Prevention ... type 2 diabetes and certain types of cancer, some of the leading causes of ...
(Date:2/8/2016)... ... 2016 , ... The Bell Agency, a full service insurance ... their ongoing community enrichment program. The current campaign fundraises for Angels & Doves, ... being accepted at: http://www.angelsanddoves.com/donate.html . , Angels & Doves was founded in ...
(Date:2/8/2016)... ... February 08, 2016 , ... ... networking and relationship-marketing firm, announced today that nominations will be accepted February ... (ISE®) West Awards. , Awards include the Information Security Executive® of ...
Breaking Medicine News(10 mins):
(Date:2/9/2016)... Mast Therapeutics, Inc. (NYSE MKT: MSTX), a biopharmaceutical ... heart failure, today announced that it intends to offer shares ... an underwritten public offering.  The offering is subject to market ... to whether or when the offering may be completed, or ...   --> --> Roth ...
(Date:2/9/2016)... 9, 2016 Hearing protection devices refer to ... transmitted to the inner ear. Hearing protection devices include ... devices are recommended for users exposed to noise levels ... to HPD that are inserted in the ear canal ... to provide more natural sound perception with the help ...
(Date:2/9/2016)... This TforG report includes the Surgical Procedures Database with ... in South Korea . To add ... written and numerical analysis on the current trends, size, ... latest reforms of the local Healthcare system are clearly ... The report helps businesses gain a unique insight into ...
Breaking Medicine Technology: