Figure 7.

Long-term engraftment of ALS20-transduced human CD34⁺ cells in immunocompromised mice

(A) Flow cytometry assessment of human CD45⁺ cells over mouse CD45⁺ cells (top) and of human CD34⁺ cells within the human CD45⁺ cells in bone marrow harvested 4 months after bone marrow transplantation (bottom). (B) VCN in bone marrow of NSG mice (n = 8) injected with human CD34⁺ cells transduced with ALS20 and collected after 4 months. (C and D) Comparison of the distributions of lentiviral vector integration sites in bone marrow specimens from the 8 animals injected with ALS20 (from A and B, coded with p and a number) relative to similar data from human stem cell gene therapy studies with safe outcomes for 6 peripheral blood mononuclear cell (PBMC) samples, 6 months after gene therapy for WAS and CGD. Samples (in columns) are compared to genomic features and epigenetic marks (in rows), respectively, as in Figure 3.