A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward ...
Nov 7, 2016
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UCLA stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell disease—the nation's most common inherited blood disorder.
Mar 9, 2015
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The ability to switch out one gene for another in a line of living stem cells has only crossed from science fiction to reality within this decade. As with any new technology, it brings with it both promise—the hope of fixing ...
Jul 9, 2014
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Genetic mutations that affect our blood cells' haemoglobin are the most common of all mutations. It has been estimated that around 5% of the world's population carry a defective globin gene.
Jul 9, 2014
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A research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center and other institutions has discovered a new genetic target for potential therapy of sickle cell disease (SCD). The target, called an enhancer, ...
Oct 10, 2013
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Researchers at Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) have taken the first preliminary steps toward developing a form of gene therapy for sickle cell disease. In an abstract presented on Dec. 10 at the 54th ...
Dec 11, 2012
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More than 60 years ago, scientists discovered the underlying cause of sickle cell disease: People with the disorder produce crescent-shaped red blood cells that clog capillaries instead of flowing smoothly, like ordinary, ...
Feb 29, 2012
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Sickle cell anemia causes pain, fatigue and delayed growth, all because of a lack of enough healthy red blood cells. And yet genetic mutations that cause it - recessive genes for the oxygen-carrying hemoglobin protein - have ...
Nov 30, 2011
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New research shows a drug commonly used to treat sickle cell anemia in adults reduces bouts of acute pain and a pneumonia-like illness, cuts hospitalization time and eases other symptoms of the disease in young patients. ...
May 12, 2011
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