Non-replicating Epstein-Barr virus-based plasmids extend gene expression and can improve gene therapy in vivo

J Biol Chem. 2000 Sep 29;275(39):30408-16. doi: 10.1074/jbc.M004782200.

Abstract

To date, no gene transfer vector has produced prolonged gene expression following a single intravenous injection and then efficiently re-expressed the delivered gene following repeated systemic injection into immunocompetent hosts. To overcome these limitations, a gene therapy regimen using non-replicating Epstein-Barr virus (EBV)-based expression plasmids was developed. One plasmid contains the FR (EBV family of repeats) sequence and the expressed gene. The other encodes Epstein-Barr nuclear antigen 1 (EBNA-1), but lacks FR. Although unable to replicate in mice, intravenous co-injection of EBV-based plasmids in cationic liposome-DNA complexes (CLDCs) substantially prolonged luciferase gene expression. The use of a two-vector system limited host exposure to the EBNA-1 gene product. Furthermore, this EBV-based vector system could be intravenously re-injected multiple times into immunocompetent mice without loss of transfection efficiency. Use of this vector system significantly improved the therapeutic efficacy of the biologically important human granulocyte colony-stimulating factor gene. Delivery of the human granulocyte colony-stimulating factor gene in EBV-based plasmids increased circulating white blood counts for at least 2 months following a single CLDC-based intravenous co-injection. Conversely, white blood counts were never elevated following injection of CLDCs lacking EBV-derived elements. Thus, this EBV-based plasmid vector system both markedly prolongs gene expression at therapeutic levels and efficiently and repeatedly re-transfects immunocompetent hosts. These properties of EBV-based plasmid vectors appear to be due, at least in part, to the documented abilities of the EBNA-1 protein both to retain FR-containing DNA intracellularly and within the nucleus and to block anti-EBNA-1 cytotoxic T cell responses.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • DNA, Viral / genetics*
  • Drug Carriers
  • Epstein-Barr Virus Nuclear Antigens / genetics
  • Female
  • Gene Expression
  • Genes, Reporter
  • Genetic Therapy / methods*
  • Granulocyte Colony-Stimulating Factor / administration & dosage*
  • Granulocyte Colony-Stimulating Factor / biosynthesis
  • Granulocyte Colony-Stimulating Factor / genetics
  • Herpesvirus 4, Human / genetics*
  • Herpesvirus 4, Human / growth & development
  • Humans
  • Injections, Intravenous
  • Liposomes
  • Luciferases / biosynthesis
  • Luciferases / genetics
  • Mice
  • Mice, Inbred Strains
  • Plasmids / genetics*
  • Repetitive Sequences, Nucleic Acid
  • Virus Replication

Substances

  • DNA, Viral
  • Drug Carriers
  • Epstein-Barr Virus Nuclear Antigens
  • Liposomes
  • Granulocyte Colony-Stimulating Factor
  • Luciferases