In vitro evaluation of a lentiviral two-step transcriptional amplification system using GAL4FF transactivator for gene therapy applications in bone repair

Gene Ther. 2018 Jul;25(4):260-268. doi: 10.1038/s41434-018-0024-9. Epub 2018 Jun 15.

Abstract

In this study, we developed a lentiviral two-step transcriptional amplification (TSTA) system expressing bone morphogenetic protein-2 (BMP-2) under the control of a GAL4FF transactivator to enhance gene expression and limit toxicity for bone repair applications. To this end human MSCs, isolated from bone marrow or adipose tissue, were transduced overnight with a LV-TSTA system (GAL4FF or GAL4vp16) expressing BMP-2 or GFP and evaluated in vitro for transduction efficiency, mean fluorescence intensity, cell viability, and BMP-2 production. FACS analysis of GFP-transduced MSCs confirmed successful transduction with the GAL4FF+GFP vector. Moreover, ELISA demonstrated abundant BMP-2 production by GAL4FF+BMP2-transduced human MSCs over a period of 8 weeks, with minimal cytotoxicity at all time points. Compared to GAL4vp16, GAL4FF was superior with respect to BMP production at 1, 2, 4, 6, and 8 weeks in BMSCs. In ASCs, GAL4FF was still associated with higher BMP-2 production at weeks 2-8, but this difference was not as prominent as in BMSCs. To our knowledge, this is the first report of GAL4FF-mediated BMP-2 production by human BMSCs and ASCs. Compared to the standard GAL4vp16TSTA vector, GAL4FF was associated with lower cytotoxicity and higher in vitro gene expression in both BMSCs and ASCs.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Bone Morphogenetic Protein 2 / biosynthesis*
  • Bone Morphogenetic Protein 2 / genetics*
  • Bone Regeneration / genetics*
  • Cell Differentiation / genetics
  • Cells, Cultured
  • DNA-Binding Proteins / genetics*
  • Female
  • Genetic Therapy / methods*
  • Humans
  • Lentivirus / genetics
  • Male
  • Mesenchymal Stem Cells / physiology*
  • Middle Aged
  • Osteoarthritis / pathology
  • Osteoarthritis / therapy
  • Saccharomyces cerevisiae Proteins / genetics*
  • Trans-Activators / genetics*
  • Transcription Factors / genetics*
  • Transcriptional Activation
  • Transduction, Genetic
  • Transfection

Substances

  • BMP2 protein, human
  • Bone Morphogenetic Protein 2
  • DNA-Binding Proteins
  • GAL4 protein, S cerevisiae
  • Gal-VP16
  • Saccharomyces cerevisiae Proteins
  • Trans-Activators
  • Transcription Factors