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. Author manuscript; available in PMC: 2021 Feb 8.
Published in final edited form as: Nat Med. 2020 Mar 16;26(4):535–541. doi: 10.1038/s41591-020-0790-y

Fig. 2 |. Therapeutic base editing in SCD patient CD34+ HSPCs.

Fig. 2 |

a, Base editing in two plerixafor-mobilized SCD CD34+ HSPC donors by A3A (N57Q)-BE3:sgRNA-1620. Two cycles of HSPC electroporation with 40 μM RNP were performed, separated by 24 hours. Base edits were measured by deep sequence analysis. b, HbF induction by HPLC analysis of edited erythroid progeny. Data are plotted as grand median. n=3 technical replicates from each SCD patient. c, Phase-contrast microscope representative image of Hoechst 33342 negative sorted enucleated erythroid progeny 30 minutes after sodium metabisulfite (MBS) treatment from unedited and A3A (N57Q)-BE3:sgRNA-1620 base edited SCD #2 CD34+ HSPCs. Yellow arrows indicate sickle forms. Scale bar 10 μm. Three technical replicates were performed. d, Quantification of sickle forms from unedited and base edited enucleated erythroid cells at 30 minutes following MBS treatment. Data are plotted as grand median. n=3 technical replicates from each SCD patient.