Regulation of the fetal hemoglobin silencing factor BCL11A

被引：43

作者：

Basak, Anindita ^{[1
,2
,3
]}

Sankaran, Vijay G. ^{[1
,2
,3
]}

机构：

[1] Boston Childrens Hosp, Manton Ctr Orphan Dis Res, Div Hematol Oncol, Boston, MA USA

[2] Harvard Med Sch, Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA USA

[3] Broad Inst MIT & Harvard, Cambridge, MA USA

来源：

COOLEY'S ANEMIA | 2016年 / 1368卷

关键词：

fetal hemoglobin; hemoglobin switching; BCL11A; thalassemia; therapy; SICKLE-CELL-DISEASE; GLOBIN GENE-EXPRESSION; BETA-THALASSEMIA; GAMMA-GLOBIN; RISK-FACTORS; BINDING REGION; INDUCTION; ENHANCER; ANEMIA; HYDROXYUREA;

D O I：

10.1111/nyas.13024

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The clinical severity of sickle cell disease and beta-thalassemia, the major disorders of beta-globin, can be ameliorated by increased production of fetal hemoglobin (HbF). Here, we provide a brief overview of the fetal-to-adult hemoglobin switch that occurs in humans shortly after birth and review our current understanding of one of the most potent known regulators of this switching process, the multiple zinc finger-containing transcription factor BCL11A. Originally identified in genome-wide association studies, multiple orthogonal lines of evidence have validated BCL11A as a key regulator of hemoglobin switching and as a promising therapeutic target for HbF induction. We discuss recent studies that have highlighted its importance in silencing the HbF-encoding genes and discuss opportunities that exist to further understand the regulation of BCL11A and its mechanism of action, which could provide new insight into opportunities to induce HbF for therapeutic purposes.

引用

页码：25 / 30

页数：6

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