Whole exome sequencing (WES) of input and human engrafted bone marrow cells
Beta-thalassemia mobilized peripheral blood HSPCs were base edited and kept in culture or engrafted in NBSGW mice. Mock-transfected cells were used as control. WES was performed on genomic DNA extracted 6 days following transfection for input samples, or extracted from the human cells isolated from xenotransplanted mice at the time of euthanization.