Guiding T lymphopoiesis from pluripotent stem cells by defined transcription factors

Cell Res. 2020 Jan;30(1):21-33. doi: 10.1038/s41422-019-0251-7. Epub 2019 Nov 15.

Abstract

Achievement of immunocompetent and therapeutic T lymphopoiesis from pluripotent stem cells (PSCs) is a central aim in T cell regenerative medicine. To date, preferentially reconstituting T lymphopoiesis in vivo from PSCs remains a practical challenge. Here we documented that synergistic and transient expression of Runx1 and Hoxa9 restricted in the time window of endothelial-to-hematopoietic transition and hematopoietic maturation stages in a PSC differentiation scheme (iR9-PSC) in vitro induced preferential generation of engraftable hematopoietic progenitors capable of homing to thymus and developing into mature T cells in primary and secondary immunodeficient recipients. Single-cell transcriptome and functional analyses illustrated the cellular trajectory of T lineage induction from PSCs, unveiling the T-lineage specification determined at as early as hemogenic endothelial cell stage and identifying the bona fide pre-thymic progenitors. The induced T cells distributed normally in central and peripheral lymphoid organs and exhibited abundant TCRαβ repertoire. The regenerative T lymphopoiesis restored immune surveillance in immunodeficient mice. Furthermore, gene-edited iR9-PSCs produced tumor-specific T cells in vivo that effectively eradicated tumor cells. This study provides insight into universal generation of functional and therapeutic T cells from the unlimited and editable PSC source.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cells, Cultured
  • Core Binding Factor Alpha 2 Subunit / genetics*
  • Core Binding Factor Alpha 2 Subunit / metabolism
  • Embryonic Stem Cells / physiology
  • Graft Rejection / immunology
  • Homeodomain Proteins / genetics*
  • Homeodomain Proteins / metabolism
  • Lymphopoiesis* / genetics
  • Mice
  • Neoplasms, Experimental / immunology
  • Pluripotent Stem Cells / physiology*
  • Receptors, Antigen, T-Cell, alpha-beta / chemistry
  • Skin Transplantation
  • T-Lymphocytes / immunology*

Substances

  • Core Binding Factor Alpha 2 Subunit
  • Homeodomain Proteins
  • Receptors, Antigen, T-Cell, alpha-beta
  • homeobox protein HOXA9