A large team of medical researchers at Novartis Biomedical Research has developed a possible treatment for people with sickle cell disease. In their project, published in the journal Science, they conducted an exhaustive ...
Researchers from multiple institutions in China have found a way to use gene editing to reactivate dormant fetal oxygen-transporting proteins in adult blood cells to potentially reverse a wide range of blood disorders.
Researchers at the National Institutes of Health have created a novel gene therapy procedure that could preserve fertility in people with sickle cell disease and other genetic blood conditions. Infertility is a high-risk ...
Oct 12, 2023
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New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).
Aug 30, 2023
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A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does ...
May 4, 2023
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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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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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A series of laboratory studies is underway in the United States to improve gene therapy worldwide for sickle cell disease, a complex and sometimes deadly heritable blood disorder that dramatically affects the structure and ...
For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...
Apr 9, 2021
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