Augmentation of myocardial transfection using TerplexDNA: A novel gene delivery system

D. G. Affleck, L. Yu, D. A. Bull, S. H. Bailey, S. W. Kim

Research output: Contribution to journalArticlepeer-review

51 Scopus citations


Gene therapy is a potential new strategy for the treatment of cardiovascular disease. The most efficacious method of gene delivery remains a key hurdle to effective gene therapy. We present the application of a novel, nonviral gene delivery system (TerplexDNA) to augment myocardial transfection. The hearts of New Zealand white rabbits were injected with reporter genes, luciferase cDNA or β-galactosidase cDNA, either as naked plasmid DNA or plasmid DNA complexed with stearyl-poly(L-lysine)-low density lipoprotein (TerplexDNA). Three day left heart myocardial cell lysates produced 44571 ± 8730 RLU (RLU = total light units/mg protein) for the TerplexDNA luciferase rabbits versus 1638 ± 567 RLU for the naked luciferase rabbits (P = 0.002). Thirty days after injection, myocardial lysates produced 677 ± 52 RLU for the TerplexDNA luciferase hearts versus 18 ± 3 RLU for the naked luciferase hearts (P = 0.002). Histologic analysis of the hearts transfected with β-galactosidade showed that TerplexDNA increased the area and depth of transfection compared with the naked plasmid DNA alone. The hearts of Sprague-Dawley rats were injected in a similar fashion and analyzed at 1, 3, 5, 10, 15, 25 and 30 days after injection. The naked luciferase injected hearts showed transient elevation of luciferase activity to day 5 but fell back to baseline levels after that time-point. The TerplexDNA luciferase injected hearts had significantly elevated luciferase activity to 30 days. The Terplex gene delivery system significantly augments myocardial transfection compared with a naked plasmid DNA system alone. The advantage in transfection efficiency appears to be related to the unique properties of the TerplexDNA carrier molecule. The TerplexDNA delivery system represents a novel means to augment transfection of the myocardium.

Original languageEnglish (US)
Pages (from-to)349-353
Number of pages5
JournalGene Therapy
Issue number5
StatePublished - 2001
Externally publishedYes


  • Direct injection
  • Gene delivery
  • Myocardium
  • TerplexDNA
  • Transfection

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics


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