Gene therapy for heart failure: Where do we stand?

Charbel Naim, Armen Yerevanian, Roger J. Hajjar

Research output: Contribution to journalArticlepeer-review

10 Scopus citations

Abstract

Advances in understanding of themolecular 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.

Original languageEnglish
Article number333
JournalCurrent Cardiology Reports
Volume15
Issue number2
DOIs
StatePublished - Feb 2013

Keywords

  • Adeno-associated vectors
  • Calcium handling
  • Cardiac gene transfer
  • Gene therapy
  • Heart failure
  • Phospholamban
  • SERCA2a
  • Sarcoplasmic reticulum

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