Navigation Links
Potential gene therapy approach to sickle cell disease highlighted at American Society of Hematology
Date:12/11/2012

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 experiments will show us if that is possible."


'/>"/>

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

Page: 1 2

Related medicine news :

1. Targeted prostate biopsy has potential to improve diagnosis of prostate cancer
2. Study identifies potential new pathway for drug development
3. US Drug Watchdog Expands Their Transvaginal Mesh Initiative Because Of The Potential Number Of Victims & They are Offering the Names of the Best Women Attorneys To Help
4. Many home couches contain potentially toxic flame retardants
5. Scripps Research Institute study points to potential new therapies for cancer and other diseases
6. A*STAR scientists identify potential drug target for inflammatory diseases including cancers
7. Researchers report potential new treatment to stop Alzheimers disease
8. ACA: More than a million women could gain access to potentially life saving tests for cancer
9. RSV study shows potential for vaccine strategies to protect babies
10. Potential new technique for anticancer radiotherapy could provide alternative to brachytherapy
11. Micro-Stents May Hold Potential to Treat Glaucoma
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/22/2014)... (PRWEB) October 22, 2014 Healthcare ... completing their continuing education at home or on-the-go ... access to 20 brand new, premium Seminar-on-Demand CE ... catalog of over 2,000 hours of ... customize their educational experience. With such a diverse ...
(Date:10/22/2014)... Lintelus, Inc., an event software provider, announced ... will be speaking on the subject of “Event Technology ... 28, at the Holiday Inn in New York City. ... as a Tech Demo at 12:45pm, to educate event ... engaging event experience. , The Annual Meetings Technology Expo ...
(Date:10/22/2014)... HealthDay Reporter , TUESDAY, Oct. 21, 2014 (HealthDay ... be showing a genetic propensity for obesity as toddlers, a ... linked to increased body fat, but the same genes in ... researchers said. At 1 year, kids with these genes ... however, these genes were linked to excessive weight gain, the ...
(Date:10/22/2014)... Tara Haelle HealthDay Reporter ... among young adult men may lead to increased blood pressure, ... cause a similar rise in blood pressure for young adult ... when young adult women drank lightly or moderately, their risk ... found. "This finding parallels studies in older adult men ...
(Date:10/22/2014)... Thompson HealthDay Reporter , MONDAY, ... Monday officially tightened guidelines for health workers treating Ebola patients, ... use of a respirator at all times. The U.S. ... tougher rules after two Dallas nurses contracted Ebola while caring ... Liberian national Thomas Eric Duncan. Nina Pham is currently being ...
Breaking Medicine News(10 mins):Health News:Top Provider of Continuing Education at Home Releases 20 New Online Courses 2Health News:Jay Cormier, Sr. Vice President of Sales for Lintelus, Speaks about Event Technology Integration at Meetings Technology Expo in NYC 2Health News:Tall, Heavy 1-Year-Olds May Be at Risk for Obesity Later, Study Finds 2Health News:Binge Drinking May Boost Blood Pressure in Young Men 2Health News:Binge Drinking May Boost Blood Pressure in Young Men 3Health News:Binge Drinking May Boost Blood Pressure in Young Men 4Health News:CDC Tightens Rules on Caring for Ebola Patients 2Health News:CDC Tightens Rules on Caring for Ebola Patients 3
... global pharmaceutical giant Novartis bagged the contract to make ... vaccines. The $54.8 million contract// was finalised on Wednesday. ... million in federal contracts to fight a possible bird-flu ... also awarded contracts on Wednesday by the U.S. Department ...
... participate in a five-year study, to evaluate the risks of ... . ,Professor Lawrie Challis, an authority on mobile ... the Department of Health and the mobile phone industry. If ... of the study, which is expected to cost ?3 million. ...
... corporate downsizing not only affects those who lose jobs ... ,'Employees who remained in work after downsizing may ... said lead author Mika Kivimaki, from the Department of ... ,'In other words, enforced redundancies may boost mental ...
... failure, says a new study. // ,Gregg Fonarow ... of over 100,000 patients hospitalised because their heart condition was ... ,They found that the fatter the person, the less likely ... researchers reported their findings in the latest issue of the ...
... world were infected by HIV in each day of the year ... total, around 410,000 to 660,000 children under the age of 15 ... ,The report added that most of the children were infected ... half of the children infected last year would die of AIDS-related ...
... are becoming an increasingly important method for detecting malaria. ... a range of other strategies for controlling the disease ... insecticide-treated bed nets, indoor spraying against malaria-carrying mosquitoes and ... ,The tests are ideal for rural areas ...
Cached Medicine News:Health News:Downsizing Affects All Workers 2Health News:New Tests Promise Rapid and Accurate Detection of Malaria 2Health News:New Tests Promise Rapid and Accurate Detection of Malaria 3
(Date:10/22/2014)... , Oct. 22, 2014  AcelRx Pharmaceuticals, Inc. ... results from the  IAP310 study have been published ... a peer-reviewed journal with broad, multidisciplinary readership. ... evaluating the safety and efficacy of Zalviso, also ... (SSTS), for the treatment of post-operative pain in ...
(Date:10/22/2014)... , Oct. 22, 2014  Regulus Therapeutics ... biopharmaceutical company leading the discovery and development ... that it has demonstrated human proof-of-concept with ... study evaluating RG-101, a wholly-owned, GalNac-conjugated anti-miR ... hepatitis C virus infection ("HCV").  Interim results ...
(Date:10/20/2014)... , Oct. 20, 2014  PneumRx, Inc. ( www.pneumrx.com ... today announced completion of enrollment in their RENEW Study, ... Study is the FDA-approved IDE pivotal trial for the ... It was anticipated that the study, which ... would take until the end of 2014 to enroll ...
Breaking Medicine Technology:AcelRx Announces Publication of Zalviso Phase 3 Abdominal Trial Results 2A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 2A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 3A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 4A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 5A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 6A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 7A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment H 8PneumRx Completes RENEW Pivotal Trial Enrollment 2PneumRx Completes RENEW Pivotal Trial Enrollment 3
... PRA International, a leading Clinical Research Organization, announces that ... Annual Partnerships in Clinical Trials 2009 to be held 4 ... Rotterdam, The Netherlands. PRA operational and therapeutic leaders are ... number 32. , On 4 November, PRA,s Christian Tucat, Senior ...
... announces results for,the three months to September 30, 2009. , , Q3 2009 Financial Highlights, ... ... ... $532 million +20%, Product sales ...
Cached Medicine Technology:PRA International Experts Present a Session at the 8th Annual Partnerships in Clinical Trials 2009 2Shire Continues to Deliver Excellent Growth From Core Products 2Shire Continues to Deliver Excellent Growth From Core Products 3Shire Continues to Deliver Excellent Growth From Core Products 4Shire Continues to Deliver Excellent Growth From Core Products 5Shire Continues to Deliver Excellent Growth From Core Products 6Shire Continues to Deliver Excellent Growth From Core Products 7Shire Continues to Deliver Excellent Growth From Core Products 8Shire Continues to Deliver Excellent Growth From Core Products 9Shire Continues to Deliver Excellent Growth From Core Products 10Shire Continues to Deliver Excellent Growth From Core Products 11Shire Continues to Deliver Excellent Growth From Core Products 12Shire Continues to Deliver Excellent Growth From Core Products 13Shire Continues to Deliver Excellent Growth From Core Products 14Shire Continues to Deliver Excellent Growth From Core Products 15Shire Continues to Deliver Excellent Growth From Core Products 16Shire Continues to Deliver Excellent Growth From Core Products 17Shire Continues to Deliver Excellent Growth From Core Products 18Shire Continues to Deliver Excellent Growth From Core Products 19Shire Continues to Deliver Excellent Growth From Core Products 20Shire Continues to Deliver Excellent Growth From Core Products 21Shire Continues to Deliver Excellent Growth From Core Products 22Shire Continues to Deliver Excellent Growth From Core Products 23Shire Continues to Deliver Excellent Growth From Core Products 24Shire Continues to Deliver Excellent Growth From Core Products 25Shire Continues to Deliver Excellent Growth From Core Products 26Shire Continues to Deliver Excellent Growth From Core Products 27Shire Continues to Deliver Excellent Growth From Core Products 28Shire Continues to Deliver Excellent Growth From Core Products 29Shire Continues to Deliver Excellent Growth From Core Products 30Shire Continues to Deliver Excellent Growth From Core Products 31Shire Continues to Deliver Excellent Growth From Core Products 32Shire Continues to Deliver Excellent Growth From Core Products 33
Binocular indirect ophthalmic lenses. General diagnosis & treatment....
... Volk's super ... biomicroscopy lenses. Examination ... Provide unprecedented fundus ... addressing maximum potential ...
... series slit lamp biomicroscopy ... of the posterior pole. ... capability, specifically addressing maximum ... fundus image resolution & ...
Volk indirect lenses. High magnification views of the posterior pole....
Medicine Products: