Fig. 2: Editing of long term haemopoietic stem cells and off target effects.

a Engraftment of edited human cord blood HSCs in NSG mice in primary and secondary transplants (n = 3 biologically independent samples). b Editing efficiencies for the cells prior to transplantation and following the primary and secondary transplants (n = 3 biologically independent samples). These cells were edited with ABEmax, due to the lag time on these experiments and this has lower editing efficiencies than the ABE8 editors, which are also reflected in the in vitro (Supplementary Fig. 2d). c Location of all potential off-target effects identified by CIRCLE-seq combined with two in silico approaches (Cas-OFFinder and CRISPOR). d Targeted sequencing through oligonucleotide capture at the top 250 sites identified, which confirmed likely low level off target editing at 70 sites. e Predictions from DeepHaem of the effects of all possible off-target edits in the non-coding genome on chromatin accessibility. f MA plots based on ATAC-seq data comparing WT vs base edited cells, showing that there were no significant differentially accessible peaks detected (DESeq2, alpha 0.05). Source data are provided as a Source Data file.