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Eur Heart J. 2016 Jun 1;37(21):1651-8. doi: 10.1093/eurheartj/ehw019. Epub 2016 Feb 27.

Gene therapy for the treatment of heart failure: promise postponed.

Author information

1
Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA Sorbonne Universités, UPMC University Paris 06, AP-HP, Institute of Cardiometabolism and Nutrition (ICAN), Pitié-Salpêtrière Hospital, F-75013 Paris, France.
2
Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA.
3
Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY 10029-6574, USA roger.hajjar@mssm.edu.

Abstract

Gene therapy has emerged as a powerful tool in targeting the molecular mechanisms implicated in heart failure. Refinements in vector technology, including the development of recombinant adeno-associated vectors, have allowed for safe, long-term, and efficient gene transfer to the myocardium. These advancements, coupled with evolving delivery techniques, have placed gene therapy as a viable therapeutic option for patients with heart failure. However, after much promise in early-phase clinical trials, the more recent larger clinical trials have shown disappointing results, thus forcing the field to re-evaluate current vectors, delivery systems, targets, and endpoints. We provide here an updated review of current cardiac gene therapy programmes that have been or are being translated into clinical trials.

KEYWORDS:

Adeno-associated vectors; Excitation–contraction coupling; Gene therapy; Heart failure; Sarcoplasmic reticulum calcium ATPase

PMID:
26922809
PMCID:
PMC4887702
DOI:
10.1093/eurheartj/ehw019
[Indexed for MEDLINE]
Free PMC Article

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