single cell transcriptome analysis of regulated transgenic GATA1 expression as a gene therapy for Diamond-Blackfan anemia

We analyzed the transcriptional effects of transgenic expression of our erythroid-specific codon-optimized GATA1 lentiviral vector in differentiating red blood cells of patients diagnosed with Diamond-Blackfan anemia. Overall design: CD34+ cells from DBA patients BCH-002 and BCH-008 were treated with hG1E-GATA1 (transgene) or HMD-GFP (control) and cultured for 10 days in erythroid differentiation media prior to collection and immediate processing for scRNA sequencing with 10x RNA 3' V3 kit (10x Genomics)