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. 2018 Aug 4;10:268–280. doi: 10.1016/j.omtm.2018.07.012

Figure 2.

Figure 2

Gene Transfer Efficiency in SCD Patient-Derived BM HSPCs Transduced with β-AS3 HS4 and β-AS3 LVs

(A) Percentages of CD34+ HSPCs that gave rise to BFU-E and CFU-GM in CFC assay. Values shown are mean ± SEM of three to five independent experiments. (B) Average VCN/cell in bulk populations of erythroblasts grown in liquid culture, and pools of BFU-E and CFU-GM derived from β-AS3 HS4- and β-AS3-transduced SCD BM HSPCs. Values shown are mean ± SEM of two to six independent experiments (n = 2 donors). *p ≤ 0.05 (unpaired t test). (C) Percentage of vector+ BFU-E and CFU-GM derived from SCD BM HSPCs transduced with β-AS3 HS4. Values shown are mean ± SEM of three to six independent experiments (n = 2 donors). ***p ≤ 0.001, ****p ≤ 0.0001 (chi-squared test). (D) Clonal analysis of VCN in BFU-E and CFU-GM colonies derived from SCD BM HSPCs transduced with β-AS3 HS4 or β-AS3. Values shown are mean ± SEM of three to six independent experiments (n = 2 donors). A higher proportion of colonies harboring >3 VCN was observed for β-AS3 LV at MOIs of 36 and 360 (p ≤ 0.001; chi-squared test).