Ex vivo expansion of human mobilized peripheral blood stem cells using epigenetic modifiers

Transfusion. 2015 Apr;55(4):864-74. doi: 10.1111/trf.12904. Epub 2014 Nov 2.

Abstract

Background: Epigenetic modifications likely control the fate of hematopoietic stem cells (HSCs). The chromatin-modifying agents (CMAs), 5-aza-2'-deoxycytidine (5azaD) and trichostatin A (TSA), have previously been shown to expand HSCs from cord blood and marrow. Here we assessed whether CMA can also expand HSCs present in growth factor-mobilized human peripheral blood (MPB).

Study design and methods: 5azaD and TSA were sequentially added to CD34+ MPB cells in the presence of cytokines, and the cells were cultured for 9 days.

Results: After culture, a 3.6 ± 0.5-fold expansion of CD34+CD90+ cells, a 10.1 ± 0.5-fold expansion of primitive colony-forming unit (CFU)-mix, and a 2.2 ± 0.5-fold expansion of long-term cobblestone-area-forming cells (CAFCs) was observed in 5azaD/TSA-expanded cells. By contrast, cells cultured in cytokines without 5azaD/TSA displayed no expansion; rather, a reduction in CD34+CD90+ cells (0.7 ± 0.1-fold) and CAFCs (0.3 ± 0.1-fold) from their initial numbers was observed. Global hypomethylation corresponding with increased transcript levels of several genes implicated in HSC self-renewal, including HOXB4, GATA2, and EZH2, was observed in 5azaD/TSA-expanded MPB cells in contrast to controls. 5azaD/TSA-expanded MPB cells retained in vivo hematopoietic engraftment capacity.

Conclusion: MPB CD34+ cells from donors can be expanded using 5azaD/TSA, and these expanded cells retain in vivo hematopoietic reconstitution capacity. This strategy may prove to be potentially useful to augment HSC numbers for patients who fail to mobilize.

Publication types

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

MeSH terms

  • Adult
  • Animals
  • Antigens, CD34 / analysis
  • Azacitidine / analogs & derivatives*
  • Azacitidine / pharmacology
  • Blood Cells / cytology
  • Blood Cells / drug effects
  • Bone Marrow Cells / cytology
  • Bone Marrow Cells / drug effects
  • Cell Count
  • Cell Division
  • Cells, Cultured
  • Chromatin Assembly and Disassembly / drug effects
  • Colony-Forming Units Assay
  • Cytokines / pharmacology
  • DNA Methylation / drug effects
  • Decitabine
  • Epigenesis, Genetic / drug effects*
  • Fetal Blood / cytology
  • Graft Survival
  • Hematopoietic Stem Cell Mobilization*
  • Hematopoietic Stem Cells / cytology
  • Hematopoietic Stem Cells / drug effects*
  • Heterografts
  • Humans
  • Hydroxamic Acids / pharmacology*
  • Immunomagnetic Separation
  • Infant, Newborn
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Peripheral Blood Stem Cell Transplantation
  • Transcription, Genetic / drug effects

Substances

  • Antigens, CD34
  • Cytokines
  • Hydroxamic Acids
  • trichostatin A
  • Decitabine
  • Azacitidine