Khosravi Mohammad Ali, Abbasalipour Maryam, Concordet Jean-Paul, Berg Johannes Vom, Zeinali Sirous, Arashkia Arash, Buch Thorsten, Karimipoor Morteza
Department of Molecular Medicine, Biotechnology Research Center, Pasteur Institute of Iran, Tehran, Iran.
Institute of Laboratory Animal Science, University of Zurich, Zurich, Switzerland.
Data Brief. 2019 Dec 11;28:104974. doi: 10.1016/j.dib.2019.104974. eCollection 2020 Feb.
The data presented in this article are related to the research article entitled as "Targeted deletion of the BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: A promising approach for gene therapy of beta-thalassemia disease " [1]. BCL11A is a master regulator of γ-globin gene silencing, and suppresses fetal hemoglobin expression by association with other γ-globin suppressors, and also interacts with human beta-globin locus control region as well as intergenic region between the Aγ and δ-globin genes to reconfigure beta-globin cluster. Thus, HbF reactivation has been proposed to be an approach for the treatment of β-thalassemia through knockout of BCL11A. Accordingly, an erythroid enhancer sequence was identified that, when inactivated, led to repression of BCL11A and induction of γ-globin in the erythroid lineage [2-7]. This article describes data that obtained from BCL11A gene enhancer modification in KU812 and KG-1 cell lines using the CRISPR-Cas9 genome editing system in order to reactivate γ-globin gene expression.
本文所呈现的数据与一篇名为《利用CRISPR-Cas9系统靶向缺失BCL11A基因以重新激活胎儿血红蛋白：一种有前景的β地中海贫血疾病基因治疗方法》的研究论文相关[1]。BCL11A是γ珠蛋白基因沉默的主要调节因子，通过与其他γ珠蛋白抑制因子结合来抑制胎儿血红蛋白的表达，并且还与人β珠蛋白基因座控制区以及Aγ和δ珠蛋白基因之间的基因间区域相互作用，以重新构建β珠蛋白基因簇。因此，通过敲除BCL11A来重新激活HbF已被提议作为一种治疗β地中海贫血的方法。相应地，已鉴定出一种红系增强子序列，当其失活时，会导致BCL11A的抑制并在红系谱系中诱导γ珠蛋白的产生[2-7]。本文描述了使用CRISPR-Cas9基因组编辑系统在KU812和KG-1细胞系中对BCL11A基因增强子进行修饰以重新激活γ珠蛋白基因表达后所获得的数据。
