Inducible long-term gene expression in brain with adeno-associated virus gene transfer

Gene Ther. 1998 Dec;5(12):1604-11. doi: 10.1038/sj.gt.3300782.

Abstract

Recombinant adeno-associated virus (rAAV) vectors hold promise for treating a number of neurological disorders due to the ability to deliver long-term gene expression without toxicity or immune response. Critical to these endeavors will be controlled expression of the therapeutic gene in target cells. We have constructed and tested a dual cassette rAAV vector carrying a reporter gene under the control of the tetracycline-responsive system and the tetracycline transactivator. Transduction in vitro resulted in stable expression from the vector that can be suppressed 20-fold by tetracycline treatment. In vivo experiments, carried out to 6 weeks, demonstrated that vector-transduced expression is sustained until doxycycline administration upon which reporter gene expression is reduced. Moreover, the suppression of vector-driven expression can be reversed by removal of the drug. These studies demonstrate long-term regulated gene expression from rAAV vectors. This system will provide a valuable approach for controlling vector gene expression both in vitro and in vivo.

Publication types

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

MeSH terms

  • Animals
  • Brain / metabolism*
  • Dependovirus / genetics*
  • Doxycycline / pharmacology
  • Gene Expression / drug effects
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • HeLa Cells
  • Humans
  • Male
  • Microinjections
  • Microscopy, Fluorescence
  • Protein Synthesis Inhibitors / pharmacology
  • Rats
  • Rats, Sprague-Dawley
  • Tetracycline / pharmacology
  • Time Factors
  • Transfection / methods*
  • Transgenes

Substances

  • Protein Synthesis Inhibitors
  • Tetracycline
  • Doxycycline