A human H1-HBB11-GFP reporter embryonic stem cell line (WAe001-A-2) generated using TALEN-based genome editing

使用基于 TALEN 的基因组编辑生成的人类 H1-HBB11-GFP 报告胚胎干细胞系 (WAe001-A-2)

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作者:Vera Alexeeva, Iraz T Aydin, Christoph Schaniel, Alec W Stranahan, Sunita L D'Souza, James J Bieker

Abstract

Hemoglobin production during mammalian development is characterized by temporal switches of the genes coding for the α- and ß-globin chains. Defects in this controlled process can lead to hemoglobinapathies such as sickle cell disease and ß-thalassemia. The ability of human embryonic stem cells (hESC) to proceed through hematopoiesis could provide a clinically useful source of red blood cells. However, hESC-derived red cells exhibit an embryonic/fetal, but not adult, mode of hemoglobin expression. The resource described here is a hESC line engineered to express a reporter from its adult globin promoter, providing a screening platform for small molecules that lead to efficient induction of adult globin.

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