Introducing Genes to the Heart: All About Delivery

Circ Res. 2017 Jan 6;120(1):33-35. doi: 10.1161/CIRCRESAHA.116.310039.


Recent clinical gene therapy trials for the treatment of heart failure have failed to meet primary efficacy endpoints and have tampered enthusiasm for the future application of cardiac gene therapy. These results have brought to light the difficulty of efficiently introducing genes into the human heart and have focused on potential problems that need to be addressed before further clinical applications. These trials however have established the safety of gene delivery vectors for cardiac targeting in humans. The sinusoidal trajectory of gene therapy continues and despite these setbacks the future of the field is promising.

Keywords: adeno-associated vectors; gene therapy; heart failure; sarcoplasmic reticulum calcium ATPase.

Publication types

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

MeSH terms

  • Animals
  • Clinical Trials as Topic / methods
  • Gene Transfer Techniques / trends*
  • Genetic Therapy / methods
  • Genetic Therapy / trends*
  • Heart Failure / genetics*
  • Heart Failure / therapy*
  • Humans