Gene therapy progress and prospects: Transcription regulatory systems

Carlo Toniatti, H. Bujard, R. Cortese, G. Ciliberto

Research output: Contribution to journalArticlepeer-review


The clinical efficacy and safety as well as the application range of gene therapy will be broadened by developing systems capable of finely modulating the expression of therapeutic genes. Transgene regulation will be crucial for maintaining appropriate levels of a gene product within the therapeutic range, thus preventing toxicity. Moreover, the possibility to modulate, stop or resume transgene expression in response to disease evolution would facilitate the combination of gene therapy with more conventional therapeutic modalities. The development of ligand-dependent transcription regulatory systems is thus of great importance. Here, we summarize the most recent progress in the field.

Original languageEnglish
Pages (from-to)649-657
Number of pages9
JournalGene Therapy
Issue number8
Publication statusPublished - Apr 2004


  • Inducible transcription
  • Ligand-dependent switches
  • Transcriptional regulation

ASJC Scopus subject areas

  • Genetics


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