Gene and cell-based therapies for heart disease

Luis G. Melo, Alok S. Pachori, Deling Kong, Massimiliano Gnecchi, Kai Wang, Richard E. Pratt, Victor J. Dzau

Research output: Contribution to journalArticlepeer-review


Heart disease remains the prevalent cause of premature death and accounts for a significant proportion of all hospital admissions. Recent developments in understanding the molecular mechanisms of myocardial disease have led to the identification of new therapeutic targets, and the availability of vectors with enhanced myocardial tropism offers the opportunity for the design of gene therapies for both protection and rescue of the myocardium. Genetic therapies have been devised to treat complex diseases such as myocardial ischemia, heart failure, and inherited myopathies in various animal models. Some of these experimental therapies have made a successful transition to clinical trial and are being considered for use in human patients. The recent isolation of endothelial and cardiomyocyte precursor cells from adult bone marrow may permit the design of strategies for repair of the damaged heart. Cell-based therapies may have potential application in neovascularization and regeneration of ischemic and infarcted myocardium, in blood vessel reconstruction, and in bioengineering of artificial organs and prostheses. We expect that advances in the field will lead to the development of safer and more efficient vectors. The advent of genomic screening technology should allow the identification of novel therapeutic targets and facilitate the detection of disease-causing polymorphisms that may lead to the design of individualized gene and cell-based therapies.

Original languageEnglish
Pages (from-to)648-663
Number of pages16
JournalFASEB Journal
Issue number6
Publication statusPublished - Apr 2004


  • AAV
  • Contractile function
  • Coronary artery disease
  • Myocardial protection

ASJC Scopus subject areas

  • Agricultural and Biological Sciences (miscellaneous)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Biochemistry
  • Cell Biology


Dive into the research topics of 'Gene and cell-based therapies for heart disease'. Together they form a unique fingerprint.

Cite this