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

L.G Meloa,b, M Gnecchia, A.S Pachoria, K Wangb and V.J Dzaua,*,1

a Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
b Queen's University, Ontario, Canada

* Professor V.J. Dzau, Chairman, Department of Medicine, Brigham and Women's Hospital, Tower 1, Room 210, 75 Francis Street, Boston, MA 02115-6110, USA. Tel.: +1-617-732-6340; fax: +1-617-732-6439
vdzau{at}partners.org

Abstract

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.

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


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