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Transcriptome of erythroid cells derived from human CD34+ cells edited at the BCL11A erythroid enahncer or at the HBG1 and HBG2 promoters using CRISPR-Cas9

GSE264491 Homo sapiens Expression profiling by high throughput sequencing 9 samples Submitted 2024/12/10 Platform GPL24676
Summary
BCL11A represses gamma globin expression by binding to the gamma globin gene (HBG1 and HBG2) promoters. Genome editing of the BCL11A erythroid enhancer in the intron 2 of BCL11A gene or the BCL11A binding site at the HBG1/2 promoters disrupts this pathway and leads to gamma globin induction. Transcriptomic profiling of erythroid cells derived from human CD34+ cells edited at either target site using CRISPR-Cas9 revealed broader transcriptome perturbation when edited at the BCL11A erythroid enhancer.
Published in
Nonclinical evaluation of HBG1/2 and BCL11A as genome-editing targets for the treatment of β-hemoglobinopathies
Janoudi T, Jagdale M, Wu M et al. · Blood advances 2025 · PMID 39637306 · doi:10.1182/bloodadvances.2024014040
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Also filed as BioProject PRJNA1102703 and SRA study SRP503024. Searching any of these in the dataset finder brings you back here.

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