Tumor-targeted gene delivery: an attractive strategy to use highly active effector molecules in cancer treatment

Gene Ther. 2002 Jun;9(11):731-5. doi: 10.1038/sj.gt.3301748.

Abstract

We have developed surface-shielded ligand-polycation based gene delivery systems which are able to target gene expression to distant tumors after systemic application. Tumor-specific targeting is achieved by (1) incorporation of cell-binding ligands; and (2) shielding of the complexes from non-specific interactions with blood components and non-target cells. Shielding of polycation/DNA complexes can be achieved by coating with either polyethylene glycol or by incorporating the ligand transferrin at high densities. Following systemic application, surface-shielded DNA complexes coding for a highly active, yet highly toxic cytokine, tumor necrosis factor-alpha (TNFalpha), localized gene expression to distant tumors, resulting in hemorrhagic tumor necrosis and inhibition of tumor growth. TNFalpha activity was confined to the tumor without systemic TNF-related toxicity. These results indicate that targeted gene delivery may be an attractive strategy to use highly potent molecules in cancer treatment.

MeSH terms

  • Animals
  • Gene Expression
  • Gene Targeting / methods*
  • Genetic Engineering
  • Genetic Therapy / methods*
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred Strains
  • Neoplasms, Experimental / therapy*
  • Polyethyleneimine
  • Transferrin / genetics
  • Tumor Necrosis Factor-alpha / genetics*

Substances

  • Transferrin
  • Tumor Necrosis Factor-alpha
  • Polyethyleneimine