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:8/31/2014)... have discovered that ,good, cholesterol is turned ,bad, by ... MG, was found to damage ,good, HDL cholesterol, which ... , Low levels of HDL, High Density Lipoprotein, ... of MG being common in the elderly and those ... funding from the British Heart Foundation (BHF) and published ...
(Date:8/31/2014)... of satellite data from the last 19 years reveals that ... the coast of Antarctica to rise by 2cm more than ... of Southampton detected the rapid rise in sea-level by studying ... million square kilometres. , The melting of the ... has contributed an excess of around 350 gigatonnes of freshwater ...
(Date:8/29/2014)... SAN ANTONIO (Aug. 29, 2014) Scientists at the ... the School of Medicine at the UT Health Science ... longevity in the tissues of the longest-lived rodent, the ... factor in the cells of naked mole rats protects ... disposer for damaged and obsolete proteins. , The factor ...
Breaking Biology News(10 mins):Sugar substance 'kills' good HDL cholesterol, new research finds 2Factor in naked mole rat's cells enhances protein integrity 2
... lining , The intestines have to work properly if we ... must be secreted, the food broken down into smaller components ... muscles and organs. The lining of the gut is coated ... hormones while keeping dangerous bacteria and toxins at bay. Close ...
... August 30, 2010 The Metcalf Institute ... of Rhode Island Graduate School of Oceanography has ... RAPID Response grant through the Foundation,s office of ... Improving Communication of Oil Spill Research, will support ...
... 30th, 2010 Undergrads often take communication courses unrelated ... Department of Horticulture teamed up with the Library and ... students to research and understand literature searchers and incorporate ... Jeanine E. Aune, and Gail R. Nonnecke, Iowa State ...
Cached Biology News:Researchers to activate anti-cancer gene 2Metcalf Institute wins NSF grant to help journalism and communication of oil spill research 2Teaching communication and information literacy skills 2
(Date:8/28/2014)... Best Sanitizers, Inc., the first company ... sanitizer, asks food processors and food handling professionals to ... they’re currently using to Best Sanitizers’ Alpet® E3 ... to fighting cross-contamination and the spread of pathogens in ... are key criteria that make a hand sanitizer most ...
(Date:8/28/2014)... in spintronics, an electronic which is not only ... on their spin and the spin-related magnetism. Spin-charge ... signals and vice versa. Recently, the research group ... Physics at Johannes Gutenberg University Mainz in collaboration ... has for the first time realised a new, ...
(Date:8/28/2014)... executives from clinical trial marketing firm BBK Worldwide will lead ... Clinical Trials 2014 , to be held at the Hyatt ... Patient recruitment experts Bonnie A. Brescia , Matthew ... on the benefits of employing an effective patient engagement strategy, ... mobile apps – can be used to drive study participation ...
(Date:8/28/2014)... Aug. 28, 2014  Armetheon, Inc. ( www.armetheon.com ... mid- to late-stage cardiovascular drug candidates, today announced ... first round of financing. The Series A round ... Bioventures with participation from investors that included Atheneos ... Dr. Larry Hsu , the founder of ...
Breaking Biology Technology:Food Processors and Food Handlers are Encouraged to Prepare for Fall Harvest by Evaluating Their Current Hand Sanitizer Using Nine Key Criteria 2Food Processors and Food Handlers are Encouraged to Prepare for Fall Harvest by Evaluating Their Current Hand Sanitizer Using Nine Key Criteria 3A new, tunable device for spintronics 2A new, tunable device for spintronics 3BBK Worldwide Leads Sessions at Key September Events 2Armetheon Completes $7.0 Million Series A Financing 2Armetheon Completes $7.0 Million Series A Financing 3
... Top 25 Global Pharmaceutical Companies Select Drug ... Visualization, MOUNTAIN VIEW, Calif., April 2, 2008 ... provider of software,strategic consulting, and regulatory services for ... 25 global pharmaceutical,companies have become customers for its ...
... 1, 2008 MiMedx Group, Inc.,(OTC Bulletin Board: ... AYXC), today reported that the contemplated reverse stock ... related name change, to,MiMedx Group, Inc., was approved ... a result of the reverse split and redomestication, ...
... Installation and Major Data Migration in ... United States and Abroad, CHICAGO, April 1, ... signed an agreement with the Cleveland,Clinic, a not-for-profit ... deploy the syngo(R) Dynamics cardiology image,management and reporting ...
Cached Biology Technology:Pharsight Signs Two New DMX(R) License Customers 2Pharsight Signs Two New DMX(R) License Customers 3MiMedx Group Announces Post-split Name Change 2MiMedx Group Announces Post-split Name Change 3Siemens Signs Multi-Year Agreement With Cleveland Clinic to Deploy Enterprise-Wide Cardiology PACS 2
Rabbit polyclonal to Somatostatin 28 ( Abpromise for all tested applications). entrezGeneID: 6750 SwissProtID: P61278...
... Features Convenient platform for tube ... - 96 x 0.2ml V-bottom tubes ... Thermo-Tube Plates - Thermo-Fast® 96 Non-Skirted ... - 48 x 0.5ml Thermo-Tubes Multiple ...
... Pro upgrade for Shimadzu Phoretix 2D, Expression ... statistical tools for comparative gel analysis studies ... Data base add on component ... version 9i release 2 client software). ...
... systems provide linking and labelling reagents ... specific primary antibodies for visualising cellular ... accomplished using an ultra-sensitive indirect labelling ... technology. Secondary antibodies are polymerised directly ...
Biology Products: