Hydroxyurea down‐regulates <i>BCL11A, KLF</i>‐<i>1</i> and <i>MYB</i> through miRNA‐mediated actions to induce γ‐globin expression: implications for new therapeutic approaches of sickle cell disease

Abstract excerpt

BACKGROUND: The major therapeutic benefit of hydroxyurea, the only FDA-approved pharmacologic treatment for sickle cell disease (SCD), is directly related to fetal hemoglobin (HbF) production that leads to significant reduction of morbidity and mortality. However, potential adverse effects such as infertility, susceptibility to infections, or teratogenic effect have been subject of concerns. Therefore,...

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