Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia

<table>Pathways involved in DBA pathogenesis and possible targets for new drugs. (1) Replacement of prednisone with a GC receptor agonist with potentially less side effects, for example, deflazacort. (2) Identification of genes involved in the therapeutic response to prednisone and subsequent identification of compounds that specifically induce or activate these genes. (3) Identification of genes and pathways involved in the mechanism by which ribosomal stress induces a <i>p53</i> response. (4) Identification of genes that act downstream of p53 to induce the DBA phenotype. (5) Enhance the effect of GC in combination with other compounds, for example, stabilizers of HIF1<svg height="7.1750002" id="M11" style="vertical-align:-0.1254pt" version="1.1" viewbox="0 0 8.9375 7.1750002" width="8.9375" xmlns="http://www.w3.org/2000/svg">
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The Scientific World Journal

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Figure 1

Figure 1: Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia 