Supplementary Materials

Supplementary Material for:

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

Vittorio Sebastiano, Hanson Hui Zhen, Bahareh Haddad, Elizaveta Bashkirova, Sandra P. Melo, Pei Wang, Thomas L. Leung, Zurab Siprashvili, Andrea Tichy, Jiang Li, Mohammed Ameen, John Hawkins, Susie Lee, Lingjie Li, Aaron Schwertschkow, Gerhard Bauer, Leszek Lisowski, Mark A. Kay, Seung K. Kim, Alfred T. Lane, Marius Wernig,* Anthony E. Oro*

*Corresponding author. E-mail: wernig{at}stanford.edu (M.W.); oro{at}stanford.edu (A.E.O.)

Published 26 November 2014, Sci. Transl. Med. 6, 264ra163 (2014)
DOI: 10.1126/scitranslmed.3009540

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Immunohistochemical characterization of patient-specific iPS clones.
  • Fig. S2. GMP production of patient-specific iPS clones.
  • Fig. S3. Genetic and karyotypic characterization of patient-specific iPS clones after loop-out.
  • Fig. S4. CRISPR versus AAV-DJ targeting efficiency at the LAMA3 locus in LAMA3-deficient primary keratinocytes.
  • Fig. S5. Optimization and validation of keratinocyte differentiation protocol.
  • Fig. S6. Histology of corrected iPSC xenograft.
  • Table S1. Persistent variant genes during corrected iPS cell generation.
  • Table S2. List of genes, categorized by GO term, differentially expressed between iPS-KC and NHK.
  • References (3639)

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