Gene expression profile at single cell level of two TDT patients' PBMCs after gene editing treatment.

BCL11A encodes for a zinc-finger transcription factor that plays an important role in B-cell and pDC development. Although the +58 BCL11A enhancer we disrupted was reported as a erythroid specific enhancer, we needed to further evaluate the potential down regulation of BCL11A in other subpopulations of edited PBMCs. To study the transcriptional impact of BCL11A enhancer gene editing, we performed single-cell RNA sequencing (scRNA-seq) on PBMCs. Using microfluidics-based single-cell RNA sequencing (scRNAseq), we obtained in total 6 peripheral blood single-cell transcriptomes from 2 heathy donors (10X indicated data downloaded from 10 Genomics shared dataset), 1 patient sample before treatment and 2 patient samples after treatment. **RAW data not provided. Human data needs strict approval.**