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Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing

Persistence of human fetal hemoglobin (HbF, α(2)γ(2)) in adults lessens the severity of sickle cell disease (SCD) and the β-thalassemias. Here, we show that the repressor BCL11A is required in vivo for silencing of γ-globin expression in adult animals, yet dispensable for red cell production. BCL11A...

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Autors principals: Xu, Jian, Peng, Cong, Sankaran, Vijay G., Shao, Zhen, Esrick, Erica B., Chong, Bryan G., Ippolito, Gregory C., Fujiwara, Yuko, Ebert, Benjamin L., Tucker, Philip W., Orkin, Stuart H.
Format: Artigo
Idioma:Inglês
Publicat: 2011
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3746545/
https://ncbi.nlm.nih.gov/pubmed/21998251
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/science.1211053
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