Figure 2.

Engraftment and editing of human hematopoietic cells

Data used to generate these figures is displayed by individual mouse in Table S1. The horizontal line and the X symbol denote the mean and the median of each group, respectively.

(A) Engraftment of human hematopoietic cells in mouse bone marrow at 16-20 weeks post-injection, assessed by flow cytometry for human CD 45+ cells, and displayed as percent human CD 45+ in total marrow hematopoietic cells (human CD45⁺ and mouse CD45⁺). “Untreated” cells were from the same donor and were sham electroporated.

(B) Correction of the sickle mutation in xenografted marrow cells of individual mice, and marrow human CD34⁺ cells, at 16-20 weeks post-injection, assayed by amplicon sequencing at the HBB site and expressed as percent of HBB alleles. Input (large open circles) denotes percent correction in the pool of edited cells injected into each cohort.

(C) Indels at the HBB target site in xenografted marrow cells, and marrow CD34⁺ cells, at 16-20 weeks post-injection, assayed and displayed as for B. Input (large open circles) denotes the percent indels in the pool of edited cells injected into each cohort.