Science Advances

Supplementary Materials

This PDF file includes:

  • Fig. S1. Genome editing efficiency in erythroid cell lines.
  • Fig. S2. Erythroid maturation and γ-globin induction in HBG-edited HUDEP-2 cells.
  • Fig. S3. Cas9/gRNA RNP delivery to HSPCs leads to efficient HBG promoter editing.
  • Fig. S4. Deletion frequency at each nucleotide of the HBG promoters.
  • Fig. S5. Genome editing in stem/progenitor populations.
  • Fig. S6. Erythroid maturation and γ-globin induction in HBG-edited erythroid cells derived from SCD HSPCs.
  • Fig. S7. Off-target analysis.
  • Fig. S8. Human hematopoietic cell reconstitution in NSG mice transplanted with control and edited HSPCs.
  • Table S1. Deep sequencing analysis of edited HBG promoters in SCD HSPC-derived erythroblasts.
  • Table S2. Sanger sequencing analysis of −115-edited HBG1 and HBG2 promoters in SCD HSPC-derived erythroblasts.

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