Pre-emptive gene therapy using recombinant adeno-associated virus delivery of extracellular superoxide dismutase protects heart against ischemic reperfusion injury, improves ventricular function and prolongs survival

Gene Ther. 2004 Jun;11(12):962-9. doi: 10.1038/sj.gt.3302250.

Abstract

In high-risk patients, the ideal cardiovascular gene therapy requires a strategy that provides long-term protection of myocardium against episodes of ischemic/reperfusion injury. We report the development of an efficient, long-lasting pre-emptive gene therapy strategy in a rat model of ischemic-reperfusion (I/R) injury of heart. At 6 weeks prior to myocardial injury, the human extracellular superoxide dismutase (Ec-SOD) gene was delivered by direct intramyocardial injections, using a recombinant adeno-associated virus vector. Significant myocardial protection was documented by the decrease in infarct size at 24 h post I/R, improved left ventricular function at 7 weeks postinjury, and enhanced long-term survival in the SOD treated group. This concept of preinjury delivery and 'pre-emptive' gene therapy via the expression of a secreted protein that renders paracrine therapeutic action can be an effective strategy for organ protection against future injury.

Publication types

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

MeSH terms

  • Animals
  • Dependovirus / genetics*
  • Extracellular Fluid / enzymology
  • Genetic Therapy / methods*
  • Male
  • Myocardial Infarction / physiopathology
  • Myocardial Infarction / therapy
  • Myocardial Reperfusion Injury / mortality
  • Myocardial Reperfusion Injury / physiopathology
  • Myocardial Reperfusion Injury / prevention & control*
  • Myocardium / enzymology
  • Rats
  • Rats, Sprague-Dawley
  • Superoxide Dismutase / genetics*
  • Survival Rate
  • Time Factors
  • Transduction, Genetic / methods*
  • Ventricular Function

Substances

  • Superoxide Dismutase