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

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 i...

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Опубликовано в: :Clin Transl Med
Главные авторы: Pule, Gift Dineo, Mowla, Shaheen, Novitzky, Nicolas, Wonkam, Ambroise
Формат: Artigo
Язык:Inglês
Опубликовано: Springer Berlin Heidelberg 2016
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4824700/
https://ncbi.nlm.nih.gov/pubmed/27056246
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40169-016-0092-7
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