Supplementary Materials

Supplementary Material for:

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

Mark A. DeWitt, Wendy Magis, Nicolas L. Bray, Tianjiao Wang, Jennifer R. Berman, Fabrizia Urbinati, Seok-Jin Heo, Therese Mitros, Denise P. Muñoz, Dario Boffelli, Donald B. Kohn, Mark C. Walters, Dana Carroll*, David I. K. Martin*, Jacob E. Corn*

*Corresponding author. Email: jcorn{at} (J.E.C.); dimartin{at} (D.I.M.); dana{at} (D.C.)

Published 12 October 2016, Sci. Transl. Med. 8, 360ra134 (2016)
DOI: 10.1126/scitranslmed.aaf9336

This PDF file includes:

  • Discussion
  • Materials and Methods
  • Fig. S1. Additional data showing editing of K562 cells with the Cas9 RNP.
  • Fig. S2. Genomic context of predicted off-target cut sites for the G10 RNP.
  • Fig. S3. Additional data showing editing of CD34+ HSPCs with the Cas9 RNP.
  • Fig. S4. Qualitative detection of chromosomal translocations by overamplification PCR.
  • Fig. S5. Confirmation of efficient correction of SCD HSPCs by ddPCR.
  • Fig. S6. Additional data on engraftment and editing of HSPCs in NSG mice.
  • Table S1. Oligonucleotides used in this study.
  • Table S2. Indel mutations at cancer-associated genes in K562 cells and HSPCs edited with the trG10 RNP, as compared to unedited cells.
  • Table S3. Estimates of cellular editing in mice engrafted with edited HSPCs.
  • References (5557)

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Other Supplementary Material for this manuscript includes the following:

  • Table S4. Tabulated data from Figs. 1 to 4 (provided as an Excel file).

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