Supplementary Materials

Supplementary Material for:

Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

Christian Jörg Braun, Kaan Boztug, Anna Paruzynski, Maximilian Witzel, Adrian Schwarzer, Michael Rothe, Ute Modlich, Rita Beier, Gudrun Göhring, Doris Steinemann, Raffaele Fronza, Claudia Regina Ball, Reinhard Haemmerle, Sonja Naundorf, Klaus Kühlcke, Martina Rose, Chris Fraser, Liesl Mathias, Rudolf Ferrari, Miguel R. Abboud, Waleed Al-Herz, Irina Kondratenko, László Maródi, Hanno Glimm, Brigitte Schlegelberger, Axel Schambach, Michael Heinrich Albert, Manfred Schmidt, Christof von Kalle, Christoph Klein*

*Corresponding author. E-mail: christoph.klein@med.uni-muenchen.de

Published 12 March 2014, Sci. Transl. Med.6, 227ra33 (2014)
DOI: 10.1126/scitranslmed.3007280

This PDF file includes:

  • Fig. S1. WASP expression analysis in peripheral blood mononuclear cells in patients WAS1 to WAS10 before GT.
  • Fig. S2. WASP expression analysis in peripheral blood mononuclear cells in patients WAS1 to WAS10.
  • Fig. S3. Influence of G-CSF versus plerixafor mobilization on long-term WASP expression in leukocyte subsets.
  • Fig. S4. WASP expression analysis in platelets in patients WAS1 to WAS10.
  • Fig. S5. Immunoglobulin levels after GT.
  • Fig. S6. Diphtheria and tetanus vaccination response after GT.
  • Fig. S7. T cell proliferation analysis after GT.
  • Fig. S8. TCR Vβ spectratyping before and after GT.
  • Fig. S9. Statistical analysis of TCR Vβ spectratyping before and after GT.
  • Fig. S10. Immunological synapse formation after GT.
  • Fig. S11. NK cell lytic activity.
  • Fig. S12. Contribution of single ISs in WAS GT.
  • Fig. S13. Analysis of the IS distribution in the WAS study.
  • Fig. S14. Time course of abundance of detected insertion sites affecting already known protooncogenes and contribution of the leukemic clones.
  • Fig. S15. Analysis of lymphoid (CD3+ cells) and myeloid (neutrophils) samples regarding LMO2 and MDS1-EVI1 integrations.
  • Fig. S16. Clonality analysis to determine the heterogeneity of hematopoietic regeneration after GT by SA and SI.
  • Fig. S17. Ingenuity pathway analysis.
  • Fig. S18. Transcription analysis for the proto-oncogenes LMO2, TAL1, and LYL1.
  • Fig. S19. Longitudinal VCN analysis for patients WAS1 to WAS10.
  • Fig. S20. VCNs at the latest follow-up.
  • Fig. S21. Engraftment of leukemic clones in immunodeficient mice for patients WAS1.
  • Fig. S22. Transplantation of tumor cells in immunodeficient mice for patient WAS6.
  • Fig. S23. Transplantation of tumor cells in immunodeficient mice for patient WAS8.
  • Fig. S24. Clonality analysis for patients WAS1 and WAS5 to WAS8 after start of chemotherapy.
  • Table S1. Details of busulfan conditioning regime per patient.
  • Table S2. Transplantation characteristics.
  • Table S3. Comparison of WAS-GT features with features of Paris SCID-X1 retroviral GT.
  • Table S4. Influence of G-CSF versus plerixafor mobilization on long-term WASP expression in monocytes.
  • Table S5. T cell proliferation data.
  • Table S6. IgE levels of patients after GT.
  • Table S7. Bleeding events before and after GT.
  • Table S8. WAS score before and after GT.
  • Table S9. Outcome of HSCT.
  • Table S10. Leukemic ISs for T-ALL and AML clones.

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