Regulation of the fetal hemoglobin silencing factor BCL11A
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 …
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Dec 5, 2020 · Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor...
Genome-wide association study shows BCL11A associated with …
Correlation of the BCL11A Polymorphism with Ameliorated Homozygous β-Thalassemia in Sardinia and Effect in Sickle Cell Disease. We next explored the hypothesis that variant(s) at the BCL11A locus, by influencing the HbF levels, may modulate the …
Genome-wide association study shows BCL11A associated with
Feb 5, 2008 · These results indicate that BCL11A variants, by modulating HbF levels, act as an important ameliorating factor of the beta-thalassemia phenotype, and it is likely they could help ameliorate other hemoglobin disorders.
CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for …
Aug 4, 2022 · Gene editing to disrupt the GATA1-binding site at the +58 BCL11A erythroid enhancer could induce γ-globin expression, which is a promising therapeutic strategy to alleviate β-hemoglobinopathy...
Targeting BCL11A and KLF1 For The Treatment Of Sickle Cell …
Nov 15, 2013 · These data indicate that targeting BCL11A and/or KLF1 with ASO treatment can cause an increase in γ-globin expression that is necessary for the upregulation of fetal hemoglobin and may be used for the treatment of sickle-cell anemia and β-thalassemia.
[Advance of research on the role of BCL11A in the occurrence and ...
Apr 10, 2024 · B cell lymphoma/leukemia 11A (BCL11A) as a transcriptional repressor plays a vital role in monitoring γ/β hemoglobin switching, maintaining the normal function of hematopoietic stem cells, and regulating erythrocyte differentiation and lymphocyte development.
Post-Transcriptional Genetic Silencing of BCL11A to Treat …
Dec 5, 2020 · Uda M, Galanello R, Sanna S, et al. Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of beta-thalassemia.
BCL11A Down-Regulation Induces γ-Globin in Human β-Thalassemia …
BCL11A were down-regulated by lentiviral RNAi (RNA interference) in the K562 cell line and an in vitro culture model of human erythropoiesis in which erythroblasts are derived from the normal donor mononuclear cells (MNC) or β-TM MNC.
Targeted deletion of BCL11A gene by CRISPR-Cas9 system for …
Jul 5, 2019 · Using CRISPR-Cas9 genome-editing strategy, we deleted a 200bp genomic region within the human erythroid-specific BCL11A (B-cell lymphoma/leukemia 11A) enhancer in KU-812, KG-1, and K562 cell lines.