Navigation Links
Salk researchers develop safe way to repair sickle cell disease genes
Date:12/7/2011

LA JOLLA, CA ---- Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin.

The technique uses cells from a patient's skin to generate induced pluripotent stem cells (iPSCs), which are capable of developing into various types of mature tissues -- including blood. The scientists say their method, which repairs the beta-globin gene (HBB), avoids gene therapy techniques that can introduce potentially harmful genes into cells.

The new technique, which will soon be tested as a therapy in animals, also appears to be much more efficient than other methods tested to date, the researchers say.

"Our findings set the stage for the development of iPSC-based therapies for devastating genetic disorders such as sickle cell disease," says the study's principle investigator, Juan Carlos Izpisua Belmonte, a Salk professor and head of the Gene Expression Laboratory.

Sickle cell disease is a group of inherited blood disorders caused by genetic mutations in the HBB gene, resulting in abnormal hemoglobin, the iron-containing protein that normally allows blood cells to carry oxygen. This causes red blood cells to become hard and sticky and resemble a curved farm tool called a "sickle." In the two leading disorders caused by HBB mutations, sickle cell anemia and beta thalassemia, red blood cells can't effectively carry oxygen.

Symptoms of sickle cell disease include swelling of the hands and feet, pain due to clogging of blood vessels, anemia and stroke.

The disorders are most common among people of African, Mediterranean and Middle Eastern decent. One in every 500 African Americans and one in every 30,000 Hispanic Americans are born with sickle cell disease, according to the Centers for Disease Control and Prevention.

The disease can be cured wit
'/>"/>

Contact: Andy Hoang
ahoang@salk.edu
619-861-5811
Salk Institute
Source:Eurekalert  

Page: 1 2 3 4

Related biology news :

1. Researchers find best routes to self-assembling 3-D shapes
2. Researchers assess radioactivity released to the ocean from the Fukushima nuclear facility
3. Researchers reveal SBP8a configurations
4. Winning early-career researchers excel in entrepreneurship
5. Fraunhofer researchers receive the Franco-German Business Award 2011
6. Marine predators in trouble: UBC researchers
7. Researchers at GIS develop systematic approach for accurate DNA sequence reconstruction
8. 2 top biological imaging centers offer powerful free online tool to researchers and public
9. Adult stem cells use special pathways to repair damaged muscle, MU researchers find
10. Medical researchers in Canada and the US discover hidden side of prion diseases
11. Yale researchers develop a way to monitor engineered blood vessels as they grow in patients
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Salk researchers develop safe way to repair sickle cell disease genes
(Date:8/20/2014)... of the Para and Mato Grosso states of Brazil. ... in order to deforest the land. Deforestation is the ... the land is thereafter converted to a nonforest use. ... ranches, or urban use. The herringbone-patterned tan lines cutting ... the middle of the image are evidence of deforestation ...
(Date:8/20/2014)... Cancer Research UK scientists have discovered that acral melanomas ... Bob Marley,s death are genetically distinct from other more ... is external) published in the journal Pigment Cell & ... palms of the hands, soles of the feet, nail-beds and ... types of melanoma, it,s not caused by UV damage from ...
(Date:8/20/2014)... Wu Feng has built upon a National Science Foundation ... Cloud" program, and synergistically complemented it with subsequent collaborative ... Force on "big computing" for mini-drones and a $1 ... Health on "big data" for the life sciences. , ... each grant, he was able to tell a much ...
Breaking Biology News(10 mins):Scientists learn more about rare skin cancer that killed Bob Marley 2The internet was delivered to the masses; parallel computing is not far behind 2The internet was delivered to the masses; parallel computing is not far behind 3The internet was delivered to the masses; parallel computing is not far behind 4
... The Genetics Society of America is pleased to ... poster awards at the 51st Annual Drosophila Research Conference ... for first, second and third place awards for each ... was the first time that undergraduate student research was ...
... female zebra finches foist a part of their eggs on ... in Seewiesen discovered that in every fifth nest there is ... The female birds act in a very well-targeted way: eggs ... their own egg laying (online publication in Animal Behaviour ...
... PITTSBURGH, April 26 Regular exercise speeds learning and ... study led by researchers from the University of Pittsburgh School ... in a non-human primate model. The findings are available in ... evidence of the beneficial effects of exercise on cognition in ...
Cached Biology News:Genetics Society of America announces award recipients at 51st Annual Drosophila Conference 2Cuckoo chicks in zebra finches 2Regular aerobic exercise is good for the brain, Pitt team says 2Regular aerobic exercise is good for the brain, Pitt team says 3
(Date:8/20/2014)... , Aug. 20, 2014 Neurotrope, Inc. ... a conference call to provide an update on ... Eastern daylight timeDial-in numbers:(888) 505-4369 (U.S. and Canada) ... web cast: www.neurotropebioscience.com , under "Investor Relations"The teleconference ... completion through September 2, 2014 at (888) 203-1112 ...
(Date:8/20/2014)... 2014 The 6th Annual Arrowhead ... on October 29-30 in San Francisco, CA. This year’s ... government researchers and clinicians focused on addressing and solving ... In the twelve years since the sequencing of the ... introduced with pharmacogenetic information in the label and molecular ...
(Date:8/20/2014)... WriteResult®, a leading provider of Electronic Patient ... its myPROpad™ ePRO tablet with the addition of a ... most widely used and well-respected Patient Reported Outcome (PRO) ... is used during clinical trials and in other health ... used to demonstrate Health-Related Quality of Life outcomes (HRQoL) ...
(Date:8/19/2014)... 2014 Research and Markets  has announced the addition ... Business Report" report to their offering. This ... in US$ Thousands by the following Product Segments: Media, Sera, and ... US, Canada , Japan , ... Latin America . Annual estimates and forecasts are ...
Breaking Biology Technology:Neurotrope To Host Conference Call 26th Annual Personalized and Precision Medicine Conference Bringing Together Thought Leaders to Discuss Clinical Implementation, Dx Reimbursement, Big Data, and Sequencing 2EQ-5D™ Assessment Now Available for iPad 2Global Cell and Tissue Culture Supplies - Strategic Business Report 2014 2
... BOSTON, Jan. 11, 2012  CBI, a division of Advanstar ... 11th Annual Patient Adherence Forum, which is being held ... in Philadelphia, PA. At the event, industry experts share ... through stakeholder collaboration and patient engagement.  ...
... 11, 2012 What:  Come view ... CES, and hear Lucier and Rothberg discuss the future of ... http://photos.prnewswire.com/prnh/20110216/MM49339LOGO ) When:   Wednesday, ... Where:   Las Vegas Convention Center, North Hall, Booth ...
... 11, 2012  Inovio Pharmaceuticals, Inc. (NYSE Amex: INO ), which ... Dr. J. Joseph Kim, President and CEO, will present an overview of ... Noble Financial 8th Annual Equity Conference January ...   Inovio Presentation Tuesday, ...
Cached Biology Technology:Complete Agenda Announced for 11th Annual Patient Adherence Forum 2Inovio Pharmaceuticals to Present at Noble Financial 8th Annual Equity Conference 2Inovio Pharmaceuticals to Present at Noble Financial 8th Annual Equity Conference 3