Supplementary Materials

Supplementary Material for:

Drug discovery for Diamond-Blackfan anemia using reprogrammed hematopoietic progenitors

Sergei Doulatov, Linda T. Vo, Elizabeth R. Macari, Lara Wahlster, Melissa A. Kinney, Alison M. Taylor, Jessica Barragan, Manav Gupta, Katherine McGrath, Hsiang-Ying Lee, Jessica M. Humphries, Alex DeVine, Anupama Narla, Blanche P. Alter, Alan H. Beggs, Suneet Agarwal, Benjamin L. Ebert, Hanna T. Gazda, Harvey F. Lodish, Colin A. Sieff, Thorsten M. Schlaeger, Leonard I. Zon,* George Q. Daley*

*Corresponding author. Email: george.daley{at} (G.Q.D.); zon{at} (L.I.Z.)

Published 8 February 2017, Sci. Transl. Med. 9, eaah5645 (2017)
DOI: 10.1126/scitranslmed.aah5645

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Reprogramming of patient iPSCs.
  • Fig. S2. Hematopoietic differentiation efficiency of iPSC lines used in this study.
  • Fig. S3. In vitro characterization of DBA iPSCs.
  • Fig. S4. Characterization of CD34-5F cells derived from DBA iPSC lines.
  • Fig. S5. Chemical screening for compounds that rescue defective DBA erythropoiesis.
  • Fig. S6. Additional characterization of SMER28 activity.
  • Fig. S7. SMER28 promotes erythropoiesis through autophagy factor Atg5.
  • Table S1. Summary of normal and DBA iPSC lines used in this study.
  • Table S2. Detailed engraftment information for control and DBA CD34-5F cells.
  • Table S3. List of significant hits from the chemical library screen.

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