A type of gene therapy that precisely "edits" a key bit of DNA might offer a new way to treat sickle cell disease—a painful inherited condition that largely strikes Black children and adults.
Sep 3, 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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Research from experts at Michigan Medicine, the Children's Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy. The study ...
Aug 10, 2023
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ETH Zurich molecular biologist Mandy Boontanrart is researching gene therapies that could be used to cure two of the most common types of inherited anemia. She has now developed a promising approach for so-called beta-hemoglobinopathies. ...
Aug 2, 2023
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Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict millions of individuals across the globe.
Jun 29, 2023
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A new distributional cost-effectiveness analysis of gene therapy versus standard-of-care for sickle cell disease (SCD) found that while gene therapy is cost-ineffective by conventional measures, it can be an equitable therapeutic ...
May 29, 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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Genetic diseases can have devastating consequences for the people who inherit them. In recent years, scientists have found that there are human genetic diseases that might be treatable, and perhaps even curable, through gene ...
Apr 19, 2023
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While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps. A new study published in Blood Advances finds that the average ...
Mar 16, 2023
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People with sickle cell disease who receive nonmyeloablative stem cell transplants are at greater risk for developing new tumors, according to a new study published in the Journal of Clinical Oncology.
Mar 9, 2023
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