Periodical
HbF Inducing Eed Inhibitor Ftx-6058 Selectively and Reversibly Impacts Bone Marrow in Wild-Type Mice
العنوان: | HbF Inducing Eed Inhibitor Ftx-6058 Selectively and Reversibly Impacts Bone Marrow in Wild-Type Mice |
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المؤلفون: | Fitz, Emily M, Allen, Allysa, Edwards, Lynn, Raghunathan, Suchi, Steward, Briana, Matson, David J, Sartain, Caroline V, Bruno, Paul A, Jacobs, Jeffrey W, Stuart, Billy |
المصدر: | Blood; November 2023, Vol. 142 Issue: 1, Number 1 Supplement 1 p5265-5265, 1p |
مستخلص: | Sickle cell disease (SCD) is a genetic disorder characterized by red blood cell sickling, hemolysis, vaso-occlusive crises (VOCs), and other complications resulting from a mutation in the HBB gene. Increasing fetal hemoglobin (HbF) levels has shown promise in mitigating disease-related pathophysiology. In individuals with SCD who exhibit hereditary persistence of fetal hemoglobin (HPFH) and have HbF levels ranging from 20% to 30%, a remarkable reduction in symptoms is observed, highlighting the protective effect of increased HbF in SCD. |
قاعدة البيانات: | Supplemental Index |
تدمد: | 00064971 15280020 |
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DOI: | 10.1182/blood-2023-190115 |