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Progress in Gene Therapy for Heart Failure

Yerevanian, Armen MD; Yerevanian, Alexan BS; Hajjar, Roger J. MD

Journal of Cardiovascular Pharmacology: February 2014 - Volume 63 - Issue 2 - p 95–106
doi: 10.1097/FJC.0b013e3182a2e8b8
Invited Review Article

Abstract: Recent advances in our understanding of the pathophysiology of myocardial dysfunction in the setting of congestive heart failure have created a new opportunity in developing nonpharmacological approaches to treatment. Gene therapy has emerged as a powerful tool in targeting the molecular mechanisms of disease by preventing the ventricular remodeling and improving bioenergetics in heart failure. Refinements in vector technology, including the creation of recombinant adeno-associated viruses, have allowed for safe and efficient gene transfer. These advancements have been coupled with evolving delivery methods that include vascular, pericardial, and direct myocardial approaches. One of the most promising targets, SERCA2a, is currently being used in clinical trials. The recent success of the Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease phase 2 trials using adeno-associated virus 1-SERCA2a in improving outcomes highlights the importance of gene therapy as a future tool in treating congestive heart failure.

Cardiovascular Research Center, Mount Sinai School of Medicine, New York, NY.

Reprints: Roger J. Hajjar, MD, Cardiovascular Research Center, Mount Sinai School of Medicine, One Gustave L. Levy Place, Box 1030, New York, NY 10029 (e-mail:

Supported by NIH R01 HL093183, HL088434, P20HL100396, a National Heart, Lung, and Blood Institute Program of Excellence in Nanotechnology Award, Contract HHSN268201000045C, and P50 HL112324.

R.J.H. is a cofounder of Celladon Corporation which is developing AAV1.SERCA2a for the treatment of heart failure. The other authors declare no conflicts of interest.

Received June 06, 2013

Accepted June 28, 2013

© 2014 by Lippincott Williams & Wilkins.