Progress in gene therapy for heart failure

Armen Yerevanian, Alexan Yerevanian, Roger J. Hajjar

Research output: Contribution to journalReview articlepeer-review

5 Scopus citations

Abstract

ABSTRACT:: Recent advances in our understanding of the pathophysiology of myocardial dysfunction in the setting of congestive heart failure have created a new opportunity in developing nonpharmacological approaches to treatment. Gene therapy has emerged as a powerful tool in targeting the molecular mechanisms of disease by preventing the ventricular remodeling and improving bioenergetics in heart failure. Refinements in vector technology, including the creation of recombinant adeno-associated viruses, have allowed for safe and efficient gene transfer. These advancements have been coupled with evolving delivery methods that include vascular, pericardial, and direct myocardial approaches. One of the most promising targets, SERCA2a, is currently being used in clinical trials. The recent success of the Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease phase 2 trials using adeno-associated virus 1-SERCA2a in improving outcomes highlights the importance of gene therapy as a future tool in treating congestive heart failure.

Original languageEnglish
Pages (from-to)95-106
Number of pages12
JournalJournal of Cardiovascular Pharmacology
Volume63
Issue number2
DOIs
StatePublished - Feb 2014

Keywords

  • Adeno-associated virus
  • Calcium cycling
  • Cardiac gene therapy
  • Gene therapy
  • Heart failure
  • Phospholamban
  • SERCA

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