Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo

Mol Ther. 2012 May;20(5):1014-21. doi: 10.1038/mt.2012.46. Epub 2012 Mar 20.

Abstract

Lentiviral vectors (LV) are widely used to stably transfer genes into target cells investigating or treating gene functions. In addition, gene transfer into early murine embryos may be improved to efficiently generate transgenic mice. We applied lentiviral gene transfer to generate a mouse model transgenic for SET binding protein-1 (Setbp1) and enhanced green fluorescent protein (eGFP). Neither transgenic founders nor their vector-positive offspring transcribed or expressed the transgenes. Bisulfite sequencing of the internal spleen focus-forming virus (SFFV) promoter demonstrated extensive methylation of all analyzed CpGs in the transgenic mice. To analyze the impact of Setbp1 on epigenetic silencing, embryonic stem cells (ESC) were differentiated into cardiomyocytes (CM) in vitro. In contrast to human promoters in LV, virally derived promoter sequences were strongly methylated during differentiation, independent of the transgene. Moreover, the commonly used SFFV promoter (SFFVp) was highly methylated with remarkable strength and frequency during hematopoietic differentiation in vivo in LV but less in γ-retroviral (γ-RV) backbones. In summary, we conclude that LV using an internal SFFVp are not suitable to generate transgenic mice or perform constitutive expression studies in differentiating cells. Choosing the appropriate promoter is also crucial to allow stable transgene expression in clinical gene therapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Carrier Proteins / genetics*
  • Cell Differentiation
  • CpG Islands / genetics
  • DNA Methylation
  • Epigenesis, Genetic
  • Founder Effect
  • Gene Silencing
  • Genes, Essential
  • Genetic Vectors*
  • Green Fluorescent Proteins / genetics
  • Humans
  • Lentivirus / genetics*
  • Mice
  • Mice, Transgenic / genetics*
  • Myocytes, Cardiac / cytology
  • Myocytes, Cardiac / metabolism
  • Promoter Regions, Genetic
  • Sequence Analysis, DNA
  • Spleen Focus-Forming Viruses / genetics*
  • Stem Cells / cytology
  • Stem Cells / metabolism*
  • Transgenes

Substances

  • Carrier Proteins
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins