Adenoviral vector design for high-level transgene expression in primitive human hematopoietic progenitors

Gene Ther. 2000 Dec;7(24):2132-8. doi: 10.1038/sj.gt.3301352.

Abstract

Adenoviral vector-mediated transient gene expression can provide new possibilities for ex vivo manipulation of quiescent hematopoietic stem cells (HSC). In order to define a suitable expression cassette for high levels of transgene expression in HSCs, we have studied the level of transgene expression in human CD34+CD38- cells using adenoviral vectors with various gene expression cassettes encoding the enhanced green fluorescence protein (EGFP) gene. CD34+ hematopoietic cells were cultured in serum-free medium with megakaryocyte growth and development factor (MGDF) alone for supporting the survival of primitive progenitors or with MGDF, c-kit ligand (KL) and flt3 ligand (FL) for inducing proliferation of primitive progenitors. With all the vectors tested, higher percentages of EGFP expressing cells were found in CD34+CD38- cells than those in CD34+CD38high cells from all donors tested. The phosphoglycerate kinase (PGK)-1 promoter was found to allow higher levels of EGFP expression than the human cytomegalovirus (HCMV) promoter in CD34+CD38- cells. Replacing the SV40 polyadenylation signal with the human beta-globin gene IVS2 and polyadenylation signal in the expression cassette (Ad5xPGK-EGFP-beta-globin) enhanced the level of EGFP expression markedly further. These results provide a guideline for the development of adenoviral vectors for gene expression in human primitive hematopoietic progenitor cells. Gene Therapy (2000) 7, 2132-2138.

MeSH terms

  • ADP-ribosyl Cyclase
  • ADP-ribosyl Cyclase 1
  • Adenoviridae / genetics*
  • Antigens, CD*
  • Antigens, CD34
  • Antigens, Differentiation
  • Cells, Cultured
  • Gene Expression
  • Genes, Regulator
  • Genetic Engineering*
  • Genetic Vectors*
  • Globins / genetics
  • Green Fluorescent Proteins
  • Hematopoietic Stem Cells / immunology
  • Hematopoietic Stem Cells / metabolism*
  • Humans
  • Luminescent Proteins / genetics
  • Membrane Glycoproteins
  • NAD+ Nucleosidase
  • Phosphoglycerate Kinase / genetics
  • Simian virus 40 / genetics
  • Time Factors
  • Transduction, Genetic*

Substances

  • Antigens, CD
  • Antigens, CD34
  • Antigens, Differentiation
  • Luminescent Proteins
  • Membrane Glycoproteins
  • Green Fluorescent Proteins
  • Globins
  • Phosphoglycerate Kinase
  • ADP-ribosyl Cyclase
  • CD38 protein, human
  • NAD+ Nucleosidase
  • ADP-ribosyl Cyclase 1