Supplementary Materials

Supplementary Material for:

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Suk See De Ravin,* Xiaolin Wu, Susan Moir, Lela Kardava, Sandra Anaya-O'Brien, Nana Kwatemaa, Patricia Littel, Narda Theobald, Uimook Choi, Ling Su, Martha Marquesen, Dianne Hilligoss, Janet Lee, Clarissa M. Buckner, Kol A. Zarember, Geraldine O'Connor, Daniel McVicar, Douglas Kuhns, Robert E. Throm, Sheng Zhou, Luigi D. Notarangelo, I. Celine Hanson, Mort J. Cowan, Elizabeth Kang, Coleen Hadigan, Michael Meagher, John T. Gray, Brian P. Sorrentino, Harry L. Malech*

*Corresponding author. E-mail: sderavin{at}niaid.nih.gov (S.S.D.R.); hmalech{at}niaid.nih.gov (H.L.M.)

Published 20 April 2016, Sci. Transl. Med. 8, 335ra57 (2016)
DOI: 10.1126/scitranslmed.aad8856

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Summary of patient and product treatment.
  • Fig. S2. Surveillance of lymphocytes, platelets, and absolute neutrophil count to monitor effects of busulfan conditioning before cell infusion in patients (P1 to P5).
  • Fig. S3. Flow cytometric evaluation for CD3 T, CD19 B, and CD56 NK cells in purified PMN.
  • Fig. S4. Analysis of T cell receptor repertoire and TRECs.
  • Fig. S5. Absolute numbers of immune cells in subjects P3 to P5 after gene therapy.
  • Fig. S6. Class-switched memory B cells.
  • Fig. S7. NK cell phenotype.
  • Fig. S8. Chest computed tomography.
  • Fig. S9. Similarity index of vector integration sites.
  • Fig. S10. Vector insulator PCR.
  • Table S1. Protocol schedule for clinical procedures and follow-up.
  • References (32, 33)

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Other Supplementary Material for this manuscript includes the following:

  • Table S2 (Microsoft Excel format). IS summary in P1 and P2.
  • Table S3 (Microsoft Excel format). Combined IS from P1 up to 30m.
  • Table S4 (Microsoft Excel format). Combined IS from P2 up to 24m.
  • Table S5 (Microsoft Excel format). Lentivector integration sites in human CD34+ cells in vitro, n = 259648.
  • Table S6 (Microsoft Excel format). Comparison of in vitro and in vivo integration sites.
  • Table S7 (.pdf format). Gene ontology KEGG PATHWAY.