Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

We have developed a therapeutic strategy for beta-hemoglobinopathies aimed at reactivating fetal hemoglobin expression in red blood cells derived from human hematopoietic stem/progenitor cells edited with CRISPR/Cas9 nucleases, cytidine or adenine base editors targeting the fetal gamma-globin promoters. Here, we report the transcriptomic changes occurring in human hematopoietic stem/progenitor cells (obtained from healthy donors) 48 h after transfection with CRISPR/Cas9 nucleases, cytidine or adenine base editors. Analysis of human hematopoietic stem/progenitors obtained from 3 dfferent healthy donors and treated with CRISPR/Cas9 nucleases, cytidine or adenine base editors.