Extended Data Fig. 7: Colony-forming ability of edited HSPCs.

a, Distribution of genotypes of methylcellulose colonies displayed in Panels B and D. Numbers of clones corresponding to each category are included in the pie chart. b, In vitro (pre-engraftment) live CD34⁺ HSPCs from healthy donors were single-cell sorted into 96-well plates containing semisolid methylcellulose media for colony forming assays. 14d post-sorting cells were analyzed for morphology. Depicted are number of colonies formed for each lineage (CFU-E = erythroid lineage; CFU-GEMM = multi-lineage; or CFU-GM = granulocyte/macrophage lineage) divided by the total number of wells available for colonies. Columns represent median ± interquartile range. N = 3 experimental replicates with a minimum of 3 96-well methylcellulose-coated plates for mock, RNP only, and WGR-GFP AAV6 treatment groups; N = 2 for AAV only and HBB-HBA1 AAV6 treatment groups. c, Percent distribution of each lineage among all colonies for each treatment for Panel B. d, As above, in vitro (pre-engraftment) live CD34⁺ β-thalassemia HSPCs were sorted into 96-well plates for colony forming assays. Depicted are number of colonies formed for each lineage (B = BFU-E and C = CFU-E (erythroid lineage); GE = CFU-GEMM (multi-lineage); or GM = CFU-GM (granulocyte/macrophage lineage)) divided by the total number of wells available for colonies. Columns represent median ± interquartile range. For Mock and RNP + AAV, N = 2 experimental replicates with a minimum of 3 96-well methylcellulose-coated plates for each treatment; N = 1 experimental replicate with 3 plates for RNP only treatment. e, Percent distribution of each lineage among all colonies for each treatment for Panel D.