Gene therapy for heart failure: where do we stand?

Curr Cardiol Rep. 2013 Feb;15(2):333. doi: 10.1007/s11886-012-0333-3.

Abstract

Advances in understanding of the molecular basis of myocardial dysfunction, together with the development of increasingly efficient gene transfer technology, has placed heart failure within reach of gene-based therapy. Multiple components of cardiac contractility, including the Beta-adrenergic system, the calcium channel cycling pathway, and cytokine mediated cell proliferation, have been identified as appropriate targets for gene therapy. The development of efficient and safe vectors such as adeno-associated viruses and polymer nanoparticles has provided an opportunity for clinical application for gene therapy. The recent successful and safe completion of a phase 2 trial targeting the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) has the potential to open a new era for gene therapy in the treatment of heart failure.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Genetic Therapy / methods*
  • Genetic Vectors
  • Heart Failure / physiopathology
  • Heart Failure / therapy*
  • Humans
  • Receptors, Adrenergic, beta / physiology
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases / physiology

Substances

  • Receptors, Adrenergic, beta
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases