Combinatorial antiangiogenic gene therapy by nonviral gene transfer using the sleeping beauty transposon causes tumor regression and improves survival in mice bearing intracranial human glioblastoma

Mol Ther. 2005 Nov;12(5):778-88. doi: 10.1016/j.ymthe.2005.07.689. Epub 2005 Sep 16.

Abstract

Glioblastoma is a fatal brain tumor that becomes highly vascularized by secreting proangiogenic factors and depends on continued angiogenesis to increase in size. Consequently, a successful antiangiogenic therapy should provide long-term inhibition of tumor-induced angiogenesis, suggesting long-term gene transfer as a therapeutic strategy. In this study a soluble vascular endothelial growth factor receptor (sFlt-1) and an angiostatin-endostatin fusion gene (statin-AE) were codelivered to human glioblastoma xenografts by nonviral gene transfer using the Sleeping Beauty (SB) transposon. In subcutaneously implanted xenografts, co-injection of both transgenes showed marked anti-tumor activity as demonstrated by reduction of tumor vessel density, inhibition or abolition of glioma growth, and increase in animal survival (P = 0.003). Using luciferase-stable engrafted intracranial gliomas, the anti-tumor effect of convection-enhanced delivery of plasmid DNA into the tumor was assessed by luciferase in vivo imaging. Sustained tumor regression of intracranial gliomas was achieved only when statin-AE and sFlt-1 transposons were coadministered with SB-transposase-encoding DNA to facilitate long-term expression. We show that SB can be used to increase animal survival significantly (P = 0.008) by combinatorial antiangiogenic gene transfer in an intracranial glioma model.

Publication types

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

MeSH terms

  • Angiogenesis Inhibitors / genetics
  • Angiogenesis Inhibitors / therapeutic use*
  • Angiostatins / genetics
  • Animals
  • Brain Neoplasms / genetics
  • Brain Neoplasms / therapy*
  • DNA Transposable Elements*
  • Endostatins / genetics
  • Gene Expression
  • Gene Transfer Techniques
  • Genetic Therapy*
  • Genetic Vectors*
  • Glioblastoma / genetics
  • Glioblastoma / therapy*
  • Humans
  • Luciferases / analysis
  • Mice
  • Mice, Nude
  • Neovascularization, Pathologic / drug therapy
  • Plasmids / genetics
  • Receptors, Vascular Endothelial Growth Factor / genetics
  • Transplantation, Heterologous
  • Transposases / genetics

Substances

  • Angiogenesis Inhibitors
  • DNA Transposable Elements
  • Endostatins
  • Angiostatins
  • Luciferases
  • Receptors, Vascular Endothelial Growth Factor
  • Transposases
  • sleeping beauty transposase, human