Gene therapy for the treatment of heart failure: promise postponed

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


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.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiogenesis Inducing Agents / therapeutic use
  • Animals
  • Calcium / metabolism
  • Calcium Channels / metabolism
  • Chemokine CXCL12 / genetics
  • Clinical Trials as Topic
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • Genetic Therapy / trends*
  • Genetic Vectors
  • Heart Failure / physiopathology
  • Heart Failure / therapy*
  • Humans
  • Mice
  • Myocardial Contraction / physiology
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases / genetics
  • Stem Cells / physiology
  • Vascular Endothelial Growth Factor A / therapeutic use


  • Angiogenesis Inducing Agents
  • CXCL12 protein, human
  • Calcium Channels
  • Chemokine CXCL12
  • Vascular Endothelial Growth Factor A
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases
  • Calcium