Gene- and cell-based therapies for cardiovascular diseases: Current status and future directions

L. G. Melo, M. Gnecchi, A. S. Pachori, K. Wang, V. J. Dzau

Research output: Contribution to journalArticlepeer-review


Recent advances in understanding the molecular and cellular basis of cardiovascular diseases, together with the availability of tools for genetic manipulation of the cardiovascular system, offer possibilities for new treatments. Gene therapies have demonstrated potential usefulness in treating complex diseases such as hypertension, atherosclerosis and myocardial ischaemia based on studies of various animal models. Some of these experimental therapies have transitioned into clinical trials to assess their safety, feasibility and therapeutic potential in humans with cardiovascular disease. The recent isolation of adult progenitor cells with the capacity to differentiate into endothelial and cardiomyocyte phenotypes has opened an exciting era of cell therapies for vascularization and repair of ischaemic tissues and injured blood vessels. Despite these significant developments, we believe that the successful translation of these experimental therapies into clinical practice will require safer and more effective vectors and delivery tools, a deeper knowledge of progenitor cell biology and, finally, the documentation of efficacy and safety through multicentre randomized trials.

Original languageEnglish
JournalEuropean Heart Journal, Supplement
Issue numberE
Publication statusPublished - Sep 2004


  • Bone marrow
  • Cardiac progenitors
  • Cellular cardiomyoplasty
  • Coronary artery disease
  • Endothelial progenitor cells
  • Heme oxygenase
  • Protective gene therapy

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine


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