Supplementary Materials

Supplementary Material for:

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

Blythe D. Sather, Guillermo S. Romano Ibarra, Karen Sommer, Gabrielle Curinga, Malika Hale, Iram F. Khan, Swati Singh, Yumei Song, Kamila Gwiazda, Jaya Sahni, Jordan Jarjour, Alexander Astrakhan, Thor A. Wagner, Andrew M. Scharenberg,* David J. Rawlings*

*Corresponding author. E-mail: andrewms{at} (A.M.S.); drawling{at} (D.J.R.)

Published 30 September 2015, Sci. Transl. Med. 7, 307ra156 (2015)
DOI: 10.1126/scitranslmed.aac5530

This PDF file includes:

  • Materials and Methods
  • Fig. S1. CCR5-megaTAL amino acid sequence.
  • Fig. S2. Spectrum of indels at CCR5 target site in human T cells after megaTAL or TALEN treatment.
  • Fig. S3. Robust transduction of human primary hematopoietic cells using AAV6.
  • Fig. S4. Sequence analyses verifying seamless HDR using AAV.CCR5.GFP donor template.
  • Fig. S5. Comparison of transfection, transduction, and HDR events using CCR5 gene–editing reagents in CD3, CD4, and CD8 T cells.
  • Fig. S6. Efficacy of IDLV delivered donor template for HDR gene editing.
  • Fig. S7. Evaluation of off-target editing by CCR5 nucleases in primary human T cells.
  • Fig. S8. TCR spectratyping demonstrates maintenance of TCR complexity in gene-edited T cells.
  • Fig. S9. Variation of HDR rates with CCR5 homology arm size.
  • Table S1. Antibody sources.
  • References (5562)

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