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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From the start of the coronavirus pandemic, there has been an attempt to use science to explain the disproportionate impact of COVID-19 on different groups through the prism of race. Data from the UK and the US suggests that ...
May 28, 2020
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For Evie Junior, living with sickle cell disease has been like running a marathon.
Nov 30, 2020
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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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St. Jude Children's Research Hospital investigators have shown that using the drug hydroxyurea to boost average fetal hemoglobin levels above 20 percent in children and teenagers with sickle cell anemia was associated with ...
Nov 9, 2017
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Roughly 50 families scattered across the world share ultra-rare variants in a particular gene. Silent for years, the inherited mutations make themselves known when patients reach the fourth decade of life. Changes in vision ...
Jun 17, 2024
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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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By completely or even partially depleting a protein called midnolin in B cells, UT Southwestern Medical Center researchers suppressed leukemia and lymphoma in a mouse model genetically prone to these cancers.
May 15, 2024
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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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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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