Extended Data Fig. 5: Viability of HSPCs post-editing. | Nature Medicine

Extended Data Fig. 5: Viability of HSPCs post-editing.

From: Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Extended Data Fig. 5: Viability of HSPCs post-editing.

HSPC viability was quantified 2-4d post-editing by flow cytometry. Depicted are the percentage of cells that stained negative for GhostRed viability dye. All cells were edited with our optimized HBB WGR vector using standard conditions (that is electroporation of Cas9 RNP + sg5, 5 K MOI of AAV, and no AAV wash at 24 h). Bars represent median ± interquartile range. WT: N = 5 for mock, N = 3 for RNP only, N = 1 for AAV only, and N = 6 for RNP + AAV treatment group; SCD: N = 2 for each treatment group with exception of RNP + AAV with N = 4; β-thal: N = 3 for mock, N = 1 for RNP only, and N = 7 for RNP + AAV treatment group.

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