Reprogramming committed murine blood cells to induced hematopoietic stem cells with defined factors

Cell. 2014 Apr 24;157(3):549-64. doi: 10.1016/j.cell.2014.04.006.

Abstract

Hematopoietic stem cells (HSCs) sustain blood formation throughout life and are the functional units of bone marrow transplantation. We show that transient expression of six transcription factors Run1t1, Hlf, Lmo2, Prdm5, Pbx1, and Zfp37 imparts multilineage transplantation potential onto otherwise committed lymphoid and myeloid progenitors and myeloid effector cells. Inclusion of Mycn and Meis1 and use of polycistronic viruses increase reprogramming efficacy. The reprogrammed cells, designated induced-HSCs (iHSCs), possess clonal multilineage differentiation potential, reconstitute stem/progenitor compartments, and are serially transplantable. Single-cell analysis revealed that iHSCs derived under optimal conditions exhibit a gene expression profile that is highly similar to endogenous HSCs. These findings demonstrate that expression of a set of defined factors is sufficient to activate the gene networks governing HSC functional identity in committed blood cells. Our results raise the prospect that blood cell reprogramming may be a strategy for derivation of transplantable stem cells for clinical application.

Publication types

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

MeSH terms

  • Animals
  • Cellular Reprogramming*
  • Hematopoietic Stem Cell Transplantation
  • Hematopoietic Stem Cells / cytology*
  • Hematopoietic Stem Cells / metabolism*
  • Homeodomain Proteins / genetics
  • Mice
  • Mice, Inbred C57BL
  • Myeloid Ecotropic Viral Integration Site 1 Protein
  • N-Myc Proto-Oncogene Protein
  • Neoplasm Proteins / genetics
  • Proto-Oncogene Proteins / genetics
  • Single-Cell Analysis
  • Transcription Factors / metabolism*
  • Transcriptome

Substances

  • Homeodomain Proteins
  • MYCN protein, mouse
  • Meis1 protein, mouse
  • Myeloid Ecotropic Viral Integration Site 1 Protein
  • N-Myc Proto-Oncogene Protein
  • Neoplasm Proteins
  • Proto-Oncogene Proteins
  • Transcription Factors