Gautam Dongre's two children in India and Pascazia Mazeze's son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain.
Dec 14, 2023
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Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new ...
Nov 16, 2023
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Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K.
Nov 16, 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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Scientists may have solved the question of why Ashkenazi Jews are significantly more susceptible to a rare genetic disorder known as Gaucher disease—and the answer may help settle the debate about whether they are less ...
Feb 6, 2023
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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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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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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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Asymptomatic sickle cell disease patients actually lack a tiny part of the genome, UNSW scientists have shown.
Apr 8, 2022
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Researchers at Beam Therapeutics have developed a redesigned base editor that shows considerable promise in directly repairing the single-base mutation that causes sickle-cell disease (SCD). Many strategies are being pursued ...
Apr 20, 2021
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