The Emerging Role of Gene Therapy in the Treatment of Cardiovascular Diseases

Joel E. Barbato, Melina R. Kibbe, Edith Tzeng*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations


Cardiovascular disease is the number one source of morbidity and mortality in the United States. Therapies directed at a variety of cardiovascular diseases have blossomed over the last several decades. The advent of gene therapy, first as an intriguing tool, and subsequently with the early successes of gene trials involving the treatment of SCID, led to the development of gene therapy as a potentially exciting and viable therapy in cardiovascular diseases. A variety of novel vector technologies and delivery systems have been developed to more efficiently deliver the gene product to the desired organ or tissue bed. Early clinical trials focused on stimulating angiogenesis. Subsequently, a number of other aspects of cardiovascular disease have been identified as potential targets for gene therapy, including the prevention of restenosis, the prevention and treatment of thrombosis, and the prevention of transplant vasculopathy. With over forty clinical human trials either completed or currently enrolling, cardiovascular gene therapy has proven to be safe and initial results suggest its efficacy.

Original languageEnglish (US)
Pages (from-to)499-545
Number of pages47
JournalCritical Reviews in Clinical Laboratory Sciences
Issue number5
StatePublished - 2003


  • Gene therapy
  • Myocardial ischemia
  • Neointimal hyperplasia
  • Restenosis
  • Thrombosis
  • Transplant vasculopathy

ASJC Scopus subject areas

  • Biochemistry, medical
  • General Biochemistry, Genetics and Molecular Biology
  • Clinical Biochemistry


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