New educational materials to help the sickle cell disease community learn about gene therapies for the disease are now available from researchers at the National Human Genome Research Institute (NHGRI), part of the National ...
Sep 28, 2022
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Bluebird Bio Inc.'s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.
Sep 19, 2022
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Researchers at the Center for Genomic Regulation (CRG) have identified a DNA sequence that is crucial for pancreatic differentiation and function—and for the first time—describe how it works.
Aug 22, 2022
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Researchers at Children's Hospital of Philadelphia (CHOP) have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells. The findings, published today in Nature Genetics, refine ...
Aug 8, 2022
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Sickle cell disease is an extremely debilitating condition that affects up to 40% of the population in African countries, with patients suffering episodes of excruciating pain, organ damage and reduced life expectancy. This ...
Jul 6, 2022
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In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children's Hospital of Philadelphia (CHOP) presented new data on an investigational therapy ...
Jun 14, 2022
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The genetic blood disorders sickle cell disease and beta-thalassemia are caused by errors in the genes for hemoglobin, a protein in red blood cells that carries oxygen from the lungs to tissues throughout the body. In utero, ...
May 26, 2022
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In a study published today in Blood Advances, researchers found that privately insured individuals with sickle cell disease (SCD) spend approximately $1.7 million on disease-related medical expenses over their lifetime. These ...
May 16, 2022
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Asymptomatic sickle cell disease patients actually lack a tiny part of the genome, UNSW scientists have shown.
Apr 8, 2022
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Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty. Treatment ...
Feb 16, 2022
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