Supplementary Materials

Supplementary Material for:

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Giulia Schiroli, Samuele Ferrari, Anthony Conway, Aurelien Jacob, Valentina Capo, Luisa Albano, Tiziana Plati, Maria C. Castiello, Francesca Sanvito, Andrew R. Gennery, Chiara Bovolenta, Rahul Palchaudhuri, David T. Scadden, Michael C. Holmes, Anna Villa, Giovanni Sitia, Angelo Lombardo, Pietro Genovese,* Luigi Naldini*

*Corresponding author. Email: genovese.pietro{at} (P.G.); naldini.luigi{at} (L.N.)

Published 11 October 2017, Sci. Transl. Med. 9, eaan0820 (2017)
DOI: 10.1126/scitranslmed.aan0820

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Humanized SCID-X1 mice.
  • Fig. S2. Phenotypical and functional characterization of humanized SCID-X1 mice.
  • Fig. S3. Hematopoietic reconstitution and functional studies of WT/SCID-X1 competitive transplants.
  • Fig. S4. Phenotypical characterization of lymphoblastic T lymphomas developing in mice transplanted without irradiation.
  • Fig. S5. Molecular and functional characterization of T lymphoma.
  • Fig. S6. Depletion of hematopoietic compartments with CD45-SAP in SCID-X1 mice.
  • Fig. S7. Development of a gene editing strategy for mouse HSPCs.
  • Fig. S8. Functionality of gene-edited lymphoid cells from transplanted mice.
  • Fig. S9. Functional validation of IL2RG-edited primary human T cells.
  • Fig. S10. Tailoring of gene editing protocol for human HSPCs.
  • Table S1. Phenotypical characterization of humanized SCID-X1 mice.
  • Table S2. Intron 1 IL2RG ZFNs off-target list.
  • Table S3. List of genomic gRNA target sequences.
  • Table S4. List of primers and probes.
  • Table S5. List of antibodies for flow cytometry.
  • References (3743)

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Other Supplementary Material for this manuscript includes the following:

  • Table S6. Raw data for Table 2 (provided as an Excel file).