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Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia

Am J Hematol. 2024-04; 
Amaliris Guerra, Nolan Hamilton, Ariel Rivera, Perry Demsko, Shuling Guo, Stefano Rivella
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Proteins, Expression, Isolation and Analysis … RAP-GRL was cloned and purified by GenScript. RAP-GRL was administered as described in the figure legends. TMPRSS6-ASO and Scramble-ASO were generated by IONIS … Get A Quote

摘要

A recently approved drug that induces erythroid cell maturation (luspatercept) has been shown to improve anemia and reduce the need for blood transfusion in non-transfusion-dependent as well as transfusion-dependent β-thalassemia (BT) patients. Although these results were predominantly positive, not all the patients showed the expected increase in hemoglobin (Hb) levels or transfusion burden reduction. Additional studies indicated that administration of luspatercept in transfusion-dependent BT was associated with increased erythropoietic markers, decreased hepcidin levels, and increased liver iron content. Altogether, these studies suggest that luspatercept may necessitate additional drugs for improved erythro... More

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