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:5/19/2015)... Sweden , May 18, 2015 ... fingerprint sensors FPC1020, FPC1025, FPC1145, FPC1155 and FPC1035 from one ... from June until and including Q3 2015 and the sensors ... . Communicated order values for 2015 hereby amount to ... for Q1 2015 of 140 MSEK and a number of ...
(Date:5/18/2015)... 18, 2015 Fingerprint Cards (FPC) has ... FPC1145, FPC1155 and FPC1035 from one if its module partners. ... including Q3 2015 and the sensors will be used by ... values for 2015 hereby amount to 740 MSEK to date. ... 140 MSEK and a number of smaller orders not separately ...
(Date:5/11/2015)... 11, 2015  Synaptics Incorporated (NASDAQ: SYNA ... today announced the appointment of Wajid Ali ... Officer, reporting to Rick Bergman , President ... Financial Officer, Kathleen Bayless , who announced ... Mr. Ali brings extensive financial management expertise to ...
Breaking Biology News(10 mins):Fingerprint Cards Receives Touch Fingerprint Sensor Order of SEK 230 Million and Raises Revenue Guidance for 2015 2Fingerprint Cards Receives Touch Fingerprint Sensor Order of SEK 230 Million and Raises Revenue Guidance for 2015 2Synaptics Appoints Wajid Ali as Senior Vice President and Chief Financial Officer 2
... Medicine and Biology (SIUMB) have agreed to a five-year partnership ... 2013 with Volume 16. Appearing quarterly, the Journal of ... be available exclusively in electronic format on SpringerLink . ... of Ultrasound deals with significant advances in the physics, ...
... is growing scientific concern that corals could retreat from equatorial ... team of international marine researchers warned today. Working on ... episode of global warming, the period between the last two ... of a sharp decline in coral diversity near the equator. ...
... sugary foods causes chemical changes in the brain, meaning that ... drug withdrawal, according to a study published today by Dr. ... and its affiliated CRCHUM Hospital Research Centre. "By working with ... own, we discovered that the neurochemistry of the animals who ...
Cached Biology News:Springer will collaborate with the Italian Society for Ultrasound in Medicine and Biology 2Scary news for corals -- from the Ice Age 2Could ending your fatty food habit cause withdrawal symptoms and depression? 2
(Date:5/28/2015)... 27, 2015 Research and Markets( http://www.researchandmarkets.com/research/l9dqwt/animal_genetics ) ... Market by Products, by Testing services - Global Forecast ... The global animal genetics market is estimated to be ... a CAGR of 8-9% during the forecast period of ... driven by increasing animal protein consumption, increasing adoption of ...
(Date:5/28/2015)... 28, 2015 Celsion Corporation (the "Company") (NASDAQ: ... commitments from two institutional healthcare investors to purchase an ... stock in an at-the-market registered direct offering and a ... The Company entered into a definitive purchase ... agreed to sell an aggregate of 3,000,000 shares of ...
(Date:5/28/2015)... NORTH BRUNSWICK, N.J. , May 28, 2015 ... that PDS0101, a cancer immunotherapy treatment targeting HPV-16, ... patients.  Currently, pre-cervical cancer is ... could offer a simple and effective non-surgical alternative. ... body,s defense mechanisms (T-cells) to recognize, target, and ...
(Date:5/28/2015)... New York and London (PRWEB) May 28, 2015 ... CFD-focused provider of Computer Aided Engineering software, announced ... Solid Research, Inc. (PSRI). CD-adapco will provide state-of-the-art ... PSRI members will share their expertise and experiences ... chemical and refining industry members and advance the ...
Breaking Biology Technology:Global Animal Genetics (Canine, Equine, Poultry, Porcine, Bovine, and Genetic Material) Market 2015 - Global Forecast to 2020 2Celsion Corporation Announces $8 Million At-The-Market Registered Direct Offering 2Celsion Corporation Announces $8 Million At-The-Market Registered Direct Offering 3PDS Biotechnology reports positive Phase I clinical trial results in proprietary HPV-Cancer Immunotherapy 2CD-adapco and PSRI Chartering New Course in Particle Flow Simulations 2
... Informex , the leading meeting place for buyers and sellers ... in the Informex Online Series, titled Beyond Manufacturing: Driving the ... international presence for the event. On December 1, 2011 at ... virtual platform that can be accessed from anywhere in the ...
...  If algae is to be the solution for America,s pain ... that a reality. Applications are now available for continuing education ... workers as general science technicians in the rapidly expanding biofuels ... Approximately 55 students will begin classes in March ...
... streaming of real-time information across your field of vision is ... prototype contact lens that could potentially provide the wearer with ... 22 November, in IOP Publishing,s Journal of Micromechanics and ... its safety by testing it on live eyes. There were ...
Cached Biology Technology:International Attendance for Networking and Education at Informex Beyond Manufacturing 2International Attendance for Networking and Education at Informex Beyond Manufacturing 3Free Job Training: 55 Grants Available to Retrain Workers for Biofuels Industry 2Free Job Training: 55 Grants Available to Retrain Workers for Biofuels Industry 3Terminator-style info-vision takes step towards reality 2
Antibody specific for histone H3 acetylated at any of the five N-terminal lysine residues. Rabbit polyclonal serum. Antigen: Synthetic peptide derived from histone H3 N-terminal region, mulitpliy...
NuParaffin Kit Application: Flow Cytometry Reactivity: Human...
...
...
Biology Products: