Topoisomerase inhibition accelerates gene expression after adeno-associated virus-mediated gene transfer to the mammalian heart

Mol Ther. 2007 Apr;15(4):764-71. doi: 10.1038/sj.mt.6300071. Epub 2007 Feb 13.

Abstract

Utility of adeno-associated virus 2 (AAV2) vectors for cardiac gene therapy is limited by the prolonged lag phase before maximal gene expression. Topoisomerase inhibition can induce AAV2-mediated gene expression in vivo, but with variable success in different tissues. In this study, we demonstrate that topoisomerase inhibition can accelerate AAV2-mediated gene expression in the mouse heart. We used an AAV2 vector expressing firefly luciferase and monitored expression kinetics using non-invasive bioluminescence imaging. In the group receiving vector alone, cardiac luciferase activity was evident from week 2 onward and increased progressively to reach a steady plateau by 9 weeks postinjection. In the group receiving vector and camptothecine (CPT), luciferase expression was evident from days 2 to 4 onward and increased rapidly to reach a steady plateau by 3-4 weeks postinjection, nearly three times faster than in the absence of CPT (P<0.05). Southern blot analysis of AAV2 genomes in cardiac tissue showed rapid conversion of the AAV2 genome from its single-stranded to double-stranded form in CPT-treated mice. Non-invasive determinations of luciferase expression correlated well with in vitro luciferase assays. Direct injection of the AAV2 vector and long-term luciferase gene expression had no detectable effects on normal cardiac function as assessed by magnetic resonance imaging.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Base Sequence
  • Camptothecin / pharmacology
  • Coronary Disease / therapy
  • DNA Primers / genetics
  • Dependovirus / genetics*
  • Enzyme Inhibitors / pharmacology
  • Gene Expression / drug effects
  • Genetic Therapy / methods
  • Genetic Vectors*
  • In Vitro Techniques
  • Luciferases, Firefly / genetics
  • Luminescent Measurements
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Myocardium / metabolism*
  • Recombinant Proteins / genetics
  • Topoisomerase I Inhibitors*
  • Transduction, Genetic

Substances

  • DNA Primers
  • Enzyme Inhibitors
  • Recombinant Proteins
  • Topoisomerase I Inhibitors
  • Luciferases, Firefly
  • Camptothecin