Novel, high expressing and antibiotic-controlled plasmid vectors designed for use in gene therapy

Gene Ther. 1996 Apr;3(4):350-6.

Abstract

The promise of effective gene therapy can only be accomplished by high-level expression and regulatable delivery of gene products. To achieve this end, a eukaryotic expression plasmid was modified to make transcription dependent on a tetracycline(Tc)-regulated chimeric transactivator. Mouse muscle injected with this two plasmid cis/trans control system expressed reporter proteins at levels five- to 10-fold greater than the cytomegalovirus immediate-early promoter-controlled parental plasmid. Tetracycline could be useful to either repress or activate transactivator-controlled expression based on the position of the tetO control sequences within the reporter plasmid. Finally, a prototype single plasmid construct was made and shown to express a self-regulating bicistronic transcript containing both the reporter and the transactivator. These Tc-controlled plasmids, termed maximum expression and regulated vectors (MERVs), have the potential to target a variety of gene therapy applications.

MeSH terms

  • Animals
  • Anti-Bacterial Agents / pharmacology
  • Base Sequence
  • Cell Line
  • Chloramphenicol O-Acetyltransferase / genetics
  • Cytomegalovirus / genetics
  • DNA, Recombinant / genetics
  • Gene Expression
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • Humans
  • Mice
  • Molecular Sequence Data
  • Muscles / metabolism
  • Plasmids / genetics*
  • Recombinant Fusion Proteins / genetics
  • Tetracycline / pharmacology
  • Trans-Activators / genetics
  • Transcriptional Activation

Substances

  • Anti-Bacterial Agents
  • DNA, Recombinant
  • Recombinant Fusion Proteins
  • Trans-Activators
  • Chloramphenicol O-Acetyltransferase
  • Tetracycline