HbF Inducing Eed Inhibitor Ftx-6058 Selectively and Reversibly Impacts Bone Marrow in Wild-Type Mice

التفاصيل البيبلوغرافية
العنوان: HbF Inducing Eed Inhibitor Ftx-6058 Selectively and Reversibly Impacts Bone Marrow in Wild-Type Mice
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
الوصف
تدمد:00064971
15280020
DOI:10.1182/blood-2023-190115