Consistent with the beta-KD reduction in >90% of beta-globin mRNA, the total area measured under the HbA peak was also reduced 11.8 fold ( p <0.01), and the total area measured under the HbF peak remained relatively unchanged with a slight increase that did not reach statistical significance.
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A synthetic model of human beta-thalassemia erythropoiesis using CD34+ cells from healthy adult donors.
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