Off-target analysis in base edited hematopoietic stem progenitor cells (HSPCs) from patients with sickle cell disease

Human sickle HSPCs (n=3 donors) were transfected using an adenine base editor and a gRNA that corrects the IVS2-1 thalassemic mutation to evaluate potential off-targets. As control, cells from each of the 3 donors was mock-transfected with TE buffer. Bulk RNAseq was performed 48h post-transfection to evaluate the impact of the editing procedure at the transcriptomic level.