Supplementary Materials

This PDF file includes:

  • Fig. S1. Selective proliferation of thEpoR-transduced HUDEP-2 cells in Epo dose de-escalation.
  • Fig. S2. Epo-free culture conditions result in no selective proliferation in an erythroleukemia cell line (K562 cells) with thEpoR transduction.
  • Fig. S3. Enhanced erythropoiesis results from thEpoR transduction in human CD34+ cells.
  • Fig. S4. Enhanced erythropoiesis of gene-modified erythroid cells with thEpoR derived from erythroid-specific promoters.
  • Fig. S5. Optimization of gene expression for both thEpoR and GFP in bicistronic lentiviral vectors.
  • Fig. S6. Greater proliferation of thEpoR-transduced CD34+ cells at early phase of erythroid differentiation.
  • Fig. S7. thEpoR-based enhanced erythropoiesis and shmiR BCL11A–based HbF induction in gene-modified human erythroid cells.
  • Fig. S8. Peripheral blood recovery after myeloablative TBI conditioning in transplanted macaques with shmiR BCL11A–only transduction.
  • Fig. S9. Stable lentiviral gene marking and reduced transgene expression in transplanted rhesus macaques with shmiR BCL11A–only transduction.
  • Fig. S10. thEpoR-mediated proliferation is allowed by either human or rhesus Epo.
  • Fig. S11. Peripheral blood recovery after myeloablative TBI conditioning in transplanted macaques with thEpoR/shmiR BCL11A transduction.
  • Fig. S12. Stable lentiviral gene marking and transgene expression in transplanted rhesus macaques with thEpoR/shmiR BCL11A transduction.
  • Fig. S13. GFP and Venus signals in the RBC fraction from transplanted macaques.
  • Fig. S14. A positive correlation between HbF induction and gene marking in macaques undergoing thEpoR/shmiR BCL11A transduction and transplantation.
  • Fig. S15. Coexpression of thEpoR allows for enhanced erythropoiesis and HbF induction with shmiR BCL11A in SCD CD34+ cells.

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