Navigation Links
Potential gene therapy approach to sickle cell disease highlighted at ASH
Date:12/11/2012

y, suggesting that strategies targeting a molecular switch called BCL11A have the potential to correct sickle cell disease.

First described over 100 years ago, sickle cell disease (or sickle cell anemia) is an inherited blood disease caused by a single mutation in one of the components of hemoglobin, the oxygen-carrying protein in red blood cells. The mutation reduces the protein's ability to carry oxygen and forces the cells to curve into a distinctive crescent or sickle shape.

Our bodies can actually manufacture two forms of hemoglobin: the adult form susceptible to the sickle cell mutation, and a fetal form that is largely produced during development and for a short time after birth. BCL11A is a transcription factor that facilitates that shift in hemoglobin production. Fetal hemoglobin expression significantly reduces the symptoms and complications of sickle cell disease because it prevents the intracellular abnormalities caused by the sickle mutation.

In their abstract, Renella, Orkin, Williams and their colleagues reported on their efforts to develop a combined lentiviral gene transfer/RNA interference approach capable of turning down BCL11A in vitro in human and mouse cells and in vivo in a mouse bone-marrow transplant model. Among their findings, the researchers documented a five- to 20-fold increase in fetal hemoglobin production in treated mice.

The team is quick to note that they have not yet attempted to correct disease in a model of sickle cell using this approach. Such efforts would be carried out in future studies.

"There are many approaches to sickle cell disease, and while we've shown we can develop viral vectors, knock down BCL11A, and induce fetal hemoglobin production, it's all been done in non-disease model systems," cautioned Renella, a fellow in the Williams laboratory at DF/CHCC. "What we don't yet know is whether we can stop production of sickled cells in a sickle cell system. Further experim
'/>"/>

SOURCE Boston Children's Hospital
Copyright©2012 PR Newswire.
All rights reserved

Page: 1 2 3

Related biology news :

1. New coronavirus has many potential hosts, could pass from animals to humans repeatedly
2. ALS TDI and Gladstone Institutes collaborate to discover potential ALS treatments
3. Fracking in Michigan: U-M researchers study potential impact on health, environment, economy
4. Potentially toxic flame retardants found in many US couches
5. Surface analysis techniques for advanced materials enhance Mazovias research potential
6. An international competition reaffirms the potential of bioinformatics in the diagnosis of disease
7. Scripps Research Institute team identifies a potential cause of Parkinsons disease
8. Researchers tap into CO2 storage potential of mine waste
9. Stem cell scientists discover potential way to expand cells for use with patients
10. King Richard III search in new phase after discovery has potential to rewrite history
11. New potential targets discovered for treating squamous cell lung cancers
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/18/2014)... BUFFALO, N.Y. A sleep-promoting circuit located deep in ... deep sleep. Discovered by researchers at Harvard School of ... and Biomedical Sciences, this is only the second "sleep ... to be both necessary and sufficient to produce deep ... Neuroscience , the study demonstrates that fully half of ...
(Date:9/18/2014)... robotic system that can operate inside the bore of ... of a biomedical research partnership program at Brigham and ... if the robot, in conjunction with real-time MRI images, ... costly, and less discomforting for the patient. The novel ... therapies with greater precision. , Developed by a team ...
(Date:9/18/2014)... MADISON, Wis. No matter how many times ... as social, communicating creatures. , But by ... single-celled organisms radically alter their behavior to suit ... many of them are present, and act accordingly. ... the millions, it may change from innocuous to ...
Breaking Biology News(10 mins):No sedative necessary: Scientists discover new 'sleep node' in the brain 2Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 2Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 3Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 4Trial begins for MRI-compatible robot designed to improve accuracy of prostate biopsies 5Researchers study vital 'on/off switches' that control when bacteria turn deadly 2Researchers study vital 'on/off switches' that control when bacteria turn deadly 3
... that BioMed Central's Open Access journals are high quality ... Central have again received impact factors that compare well ... five titles in the top five of their specialty. ... they are respected by researchers, and are fast becoming ...
... Impressive impact factors prove that BioMed Central's Open Access ... Journals published by BioMed Central have again received impact ... was announced today, with five titles in the top ... these journals affirm that they are respected by researchers, ...
... in repairing lung damage, according to a team of scientists ... repair process in lung transplant patients. , Reporting in today's ... of The University of Texas Medical School at Houston, and ... that less than 1 percent of a certain type of ...
Cached Biology News:'Bionic' arm gives amputee sense of touch 2Open Access journals get impressive impact factors 2Circulating stem cells play small role in lung repair 2Circulating stem cells play small role in lung repair 3
(Date:9/19/2014)... The new , Healthcare and Life ... today on PharmaBoardroom.com for free download , ... the Spanish economy in 2009, and the austerity measures that ... pragmatic in times of trouble. "Despite the crisis, ... European market, and the general conditions to invest are favorable ...
(Date:9/18/2014)... 18, 2014 A novel robotic system ... scanner is currently being tested as part of a ... in Boston with the aim of determining if the ... prostate cancer biopsies faster, more accurate, less costly, and ... has the potential to deliver prostate cancer therapies with ...
(Date:9/18/2014)... 18, 2014 Research and Markets ... Chinese Stem Cell Industry Report, 2014-2017" report to ... biological cells that can differentiate into specialized cells and ... Stem cell therapy can be applied to treatment of ... nervous system diseases, damage or lesion of liver, kidney ...
(Date:9/18/2014)... LOUIS , Sept. 18, 2014  U.S. biotech ... by signing an exclusive license for technology developed by ... control gene expression in plants, including for applications in ... The technology was developed under the ... officer at Kultevat and former president of the Danforth ...
Breaking Biology Technology:PharmaBoardroom Releases New Spain Pharmaceuticals Report 2PharmaBoardroom Releases New Spain Pharmaceuticals Report 3Worcester Polytechnic Institute Participates in Clinical Trial for MRI-Compatible Robot Designed to Improve Accuracy of Prostate Cancer Biopsies 2Worcester Polytechnic Institute Participates in Clinical Trial for MRI-Compatible Robot Designed to Improve Accuracy of Prostate Cancer Biopsies 3Worcester Polytechnic Institute Participates in Clinical Trial for MRI-Compatible Robot Designed to Improve Accuracy of Prostate Cancer Biopsies 4Worcester Polytechnic Institute Participates in Clinical Trial for MRI-Compatible Robot Designed to Improve Accuracy of Prostate Cancer Biopsies 5Worcester Polytechnic Institute Participates in Clinical Trial for MRI-Compatible Robot Designed to Improve Accuracy of Prostate Cancer Biopsies 6Global and Chinese Stem Cell Industry Report, 2014-2017 2Global and Chinese Stem Cell Industry Report, 2014-2017 3Kultevat obtains license of gene switch technology 2
... suffering from uterine fibroids, a debilitating condition that affects ... soon have reason for hope.  Halt Medical, a biomedical ... developing a minimally invasive procedure for treating the condition. ... Latin America at the NIH Global Fibroid Congress.   ...
... One of the requirements to keep trends in computer technology ... energy-efficient is faster writing and processing of data. In ... physicists at the Technische Universitaet Muenchen (TUM) and the Universitaet ... a solution. TUM physicists set a lattice of magnetic vortices ...
... 17, 2010 InterMune, Inc. (Nasdaq: ITMN ... to prepare for the commercialization of Esbriet™ (pirfenidone) in ... a positive CHMP opinion that is now awaiting ratification ... Chief Executive Officer and President of InterMune, said, "We ...
Cached Biology Technology:Halt Medical Presents Strong Results of International Fibroid Studies at National Institutes of Health Global Congress 2Electric current moves magnetic vortices 2InterMune Announces Senior Leadership Appointments to Prepare for Launch of Esbriet™ (Pirfenidone) in Europe 2InterMune Announces Senior Leadership Appointments to Prepare for Launch of Esbriet™ (Pirfenidone) in Europe 3InterMune Announces Senior Leadership Appointments to Prepare for Launch of Esbriet™ (Pirfenidone) in Europe 4InterMune Announces Senior Leadership Appointments to Prepare for Launch of Esbriet™ (Pirfenidone) in Europe 5InterMune Announces Senior Leadership Appointments to Prepare for Launch of Esbriet™ (Pirfenidone) in Europe 6
... raised against a partial recombinant ... ZFHX4 (NP_078997, 2 a.a. ... protein with GST tag. ... NM_024721 Protein ...
EPA II Buffer is a diluent formulated to stabilize the activity of Chemicon horseradish peroxidase (HRP)-antibody conjugates at high conjugate dilution for extended periods....
... can design custom microarrays to detect alternative ... Proprietary algorithms are used to identify ... high quality, spliced cDNAs aligned to the ... a custom SpliceArray is profiled with a ...
... DNA Visualizer DX's sensitivity of DNA detection ... DNA band in an agarose gel can be ... this kit can be purified by organic solvent ... tested commercially kits worked well.) DNA excised ...
Biology Products: