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资源简介：
Gene editing on the BCL11A binding site on the HBG promoter to up-regulate the gamma-globin expression
应用场景：
创建时间：
2019-07-22
相关数据集
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies [4C]
We have identified putative repressor region (PRR) to exon-1 of β-globin (βE1) in the β-globin cluster as a core region in alll the HPFH deletions that activates fetal hemoglobin and silences adult he
NIAID Data Ecosystem60
Reactivation of Zeta-Globin by deletion of BCL11A and LRF in murine cells. Reactivation of Zeta-Globin by deletion of BCL11A and LRF in murine cells
The alpha and beta-globin loci harbour genes that are expressed during development and silenced throughout post-natal life. Learning to reactivate these genes may offer new therapeutic approaches for
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In vivo base editing by a single intravenous vector injection for the treatment of hemoglobinopathies
Individuals with beta-thalassemia or Sickle Cell Disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% HbF appear to be symptom-free. Here, we used a non-integrating HDAd5/35++
NIAID Data Ecosystem10
Increasing the efficiency and targeting scope of cytidine base editors through fusion of a single-strand DNA binding protein domain
Cytidine base editors (CBEs) are powerful genetic tools which catalyze cytidine to thymidine conversion at specific genomic DNA, and further improvement of the editing scope and efficiency is critical
NIAID Data Ecosystem30
Natural regulatory mutations elevate fetal globin via disruption of BCL11A or ZBTB7A binding. Homo sapiens
B-hemoglobinopathies such as Sickle Cell Disease (SCD) and b-thalassemia result from mutations in the adult b-globin gene. Reactivating the developmentally silenced fetal g-globin gene is a therapeuti
NIAID Data Ecosystem20
