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:4/8/2015)... N.C. , April 8, 2015  Infinisource, ... solutions and services, and Morpho, a leading supplier ... partnership to produce the market,s most advanced biometrically-enabled ... Infinisource,s iSolved NXG time clock is setting a ... and labor data for the small and mid-size ...
(Date:4/2/2015)... , April 2, 2015 At its 2015 ... Lake City , the American College of Medical ... new directors to its Board.  Members of the ACMG ... and for forming and advancing its policies and programs. ... profession. "It,s an eventful time in medical ...
(Date:4/1/2015)... , Apr. 1, 2015  NXT-ID, Inc. (NASDAQ: ... authentication company focused on the growing mobile commerce market, ... Wocket smart wallets is underway to early access pre-order ... group includes usage at retail outlets including Walmart, Target, ... Users report Wocket was accepted at all outlets and ...
Breaking Biology News(10 mins):iSolved Time Clock Offers Unmatched Accuracy due to Morpho Biometrics 2iSolved Time Clock Offers Unmatched Accuracy due to Morpho Biometrics 3American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 2American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 3American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 4American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 5American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 6American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 7American College of Medical Genetics and Genomics Announces New Board Members: Dr. Louanne Hudgins is ACMG President-Elect 8NXT-ID Reports Second Shipment of Wocket Smart Wallets Underway to Early Access Pre-order Customers and Provides User Feedback 2NXT-ID Reports Second Shipment of Wocket Smart Wallets Underway to Early Access Pre-order Customers and Provides User Feedback 3NXT-ID Reports Second Shipment of Wocket Smart Wallets Underway to Early Access Pre-order Customers and Provides User Feedback 4
... responses to similar situations change as they age, according ... that combined brain scanning, eye-tracking and behavioral measures to ... Both preschool children and adults distinguish between damage ... perpetrator had done something wrong. Nonetheless, adults are much ...
... Researchers of the Max Delbrck Center for Molecular ... cells to differentiate into diverse cell types and thus ... molecule E-cadherin hitherto primarily known for its ... "intracellular glue". If E-cadherin is absent, the stem cells ...
... patients in the study cohort receiving anti-tumour necrosis factor ... first cancer within nine years and overall risk was ... nine year follow-up study conducted at Gentofte University Hospital, ... was not increased in patients treated with anti-TNFs compared ...
Cached Biology News:Changes in brain circuitry play role in moral sensitivity as people grow up 2Changes in brain circuitry play role in moral sensitivity as people grow up 3MDC researchers discover key molecule for stem cell pluripotency 2Arthritis patients taking newer treatments do not have an overall increased cancer risk 2
(Date:4/30/2015)... , April 30, 2015  Pfenex Inc. (NYSE ... the development of biosimilar therapeutics including high value ... closing of its previously announced underwritten public offering ... a public offering price of $15.50 per share. ... underwriters of their option to purchase up to ...
(Date:4/30/2015)... FRANCE (PRWEB) April 30, 2015 ... and at http://www.lespausa.com , Fleur’s ... multi-action product combining the benefits of makeup, skin ... This subtly tinted cream is designed with specific ... for an even complexion and a healthy glow. ...
(Date:4/30/2015)... (PRWEB) April 30, 2015 Sterlitech ... ultrafiltration membranes, the PX, PY, and PZ ... sheets, and disks. Designed to be ideal ... new membranes can fill critical process requirements where ... downstream processes. , “New regulations on industrial wastewater ...
(Date:4/29/2015)... --  Doximity , the leading online medical network with ... announced a new, instant method for physicians to apply ... click, all US physicians can quickly submit their verified ... open job opportunity, and a recruiter will reach out ... of physicians are open to new jobs, and yet ...
Breaking Biology Technology:Pfenex Inc. Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters' Option to Purchase Additional Shares 2Fleur’s Introduces CC CRÈME Perfect Skin Solution 2Fleur’s Introduces CC CRÈME Perfect Skin Solution 3Sterlitech Offers New Ultrafiltration Membranes 2Doximity Offering Hospitals Easy, Efficient Physician Application Tool 2
... Jan. 3 Chimerix, Inc., a,biotechnology company developing ... M.B., Ch.B., FRCA, FFPM has joined the,company as ... Dr. Frazer served as the Chief Medical,Officer and ... where he was responsible for the design of ...
... Inc. (Nasdaq: ONXX ) today announced that ... Monday, January 7, at 11:30 a.m.,Pacific Time. Interested ... at:, http://www.metameetings.com/webcasts/jpmorgan/healthcare08 , It is ... in order to,register and download any necessary software. ...
... Inc. (Nasdaq:,CEPH) today announced that it has submitted ... and Drug Administration (FDA) requesting approval of,TREANDA(R) (bendamustine ... indolent B-cell non-Hodgkin,s lymphoma (NHL) who have progressed,during ... According to the National Cancer Institute, an estimated ...
Cached Biology Technology:Chimerix Appoints Neil Frazer, M.B., Ch.B., FRCA, FFPM as Chief Medical Officer 2Cephalon Submits New Drug Application for TREANDA for the Treatment of Patients with Relapsed Indolent Non-Hodgkin's Lymphoma 2Cephalon Submits New Drug Application for TREANDA for the Treatment of Patients with Relapsed Indolent Non-Hodgkin's Lymphoma 3Cephalon Submits New Drug Application for TREANDA for the Treatment of Patients with Relapsed Indolent Non-Hodgkin's Lymphoma 4
Bethyl Laboratories packages antibodies, conjugates and calibrators to provide quantitative ELISA kits. Each kit contains the following components, sufficient for 1000 single well assays....
Bethyl Laboratories packages antibodies, conjugates and calibrators to provide quantitative ELISA kits. Each kit contains the following components, sufficient for 1000 single well assays....
Bethyl Laboratories packages antibodies, conjugates and calibrators to provide quantitative ELISA kits. Each kit contains the following components, sufficient for 1000 single well assays....
Bethyl Laboratories packages antibodies, conjugates and calibrators to provide quantitative ELISA kits. Each kit contains the following components, sufficient for 1000 single well assays....
Biology Products: