Inducible Gata1 suppression expands megakaryocyte-erythroid progenitors from embryonic stem cells

J Clin Invest. 2015 Jun;125(6):2369-74. doi: 10.1172/JCI77670. Epub 2015 May 11.

Abstract

Transfusion of donor-derived platelets is commonly used for thrombocytopenia, which results from a variety of clinical conditions and relies on a constant donor supply due to the limited shelf life of these cells. Embryonic stem (ES) and induced pluripotent stem (iPS) cells represent a potential source of megakaryocytes and platelets for transfusion therapies; however, the majority of current ES/iPS cell differentiation protocols are limited by low yields of hematopoietic progeny. In both mice and humans, mutations in the gene-encoding transcription factor GATA1 cause an accumulation of proliferating, developmentally arrested megakaryocytes, suggesting that GATA1 suppression in ES and iPS cell-derived hematopoietic progenitors may enhance megakaryocyte production. Here, we engineered ES cells from WT mice to express a doxycycline-regulated (dox-regulated) shRNA that targets Gata1 transcripts for degradation. Differentiation of these cells in the presence of dox and thrombopoietin (TPO) resulted in an exponential (at least 10¹³-fold) expansion of immature hematopoietic progenitors. Dox withdrawal in combination with multilineage cytokines restored GATA1 expression, resulting in differentiation into erythroblasts and megakaryocytes. Following transfusion into recipient animals, these dox-deprived mature megakaryocytes generated functional platelets. Our findings provide a readily reproducible strategy to exponentially expand ES cell-derived megakaryocyte-erythroid progenitors that have the capacity to differentiate into functional platelet-producing megakaryocytes.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Anti-Bacterial Agents / pharmacology*
  • Cell Differentiation / drug effects*
  • Cell Differentiation / genetics
  • Doxycycline / pharmacology*
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / metabolism*
  • GATA1 Transcription Factor / biosynthesis*
  • GATA1 Transcription Factor / genetics
  • Gene Expression Regulation / drug effects
  • Gene Expression Regulation / genetics
  • Humans
  • Megakaryocyte-Erythroid Progenitor Cells / cytology
  • Megakaryocyte-Erythroid Progenitor Cells / metabolism*
  • Megakaryocytes / cytology
  • Megakaryocytes / metabolism
  • Mice
  • Thrombopoietin / pharmacology*

Substances

  • Anti-Bacterial Agents
  • GATA1 Transcription Factor
  • Gata1 protein, mouse
  • Thrombopoietin
  • Doxycycline

Associated data

  • GEO/GSE66479