Adeno-associated virus-mediated gene therapy in cardiovascular disease

Nadjib Hammoudi, Kiyotake Ishikawa, Roger J. Hajjar

Research output: Contribution to journalReview articlepeer-review

36 Scopus citations


Purpose of review The use of adeno-associated virus (AAV) as an efficient, cardiotropic, and safe vector, coupled with the identification of key molecular targets, has placed gene-based therapies within reach of cardiovascular diseases. The purpose of this review is to provide a focused update on the current advances related to AAV-mediated gene therapy in cardiovascular diseases, and particularly in heart failure (HF), wherein gene therapy has recently made important progress. Recent findings Multiple successful preclinical studies suggest a potential utility of AAV gene therapy for arrhythmias and biological heart pacing, as well as RNA overexpression. Moreover, AAV-mediated overexpression of several molecular targets involved in HF has demonstrated promising results in clinically relevant large animal models. In humans, a safe and successful completion of a phase 2 clinical trial targeting the sarcoplasmic reticulum calcium ATPase pump with AAV has been reported. Serial studies are ongoing to further prove the efficacy of AAV-mediated sarcoplasmic reticulum calcium ATPase pump gene transfer in human HF. Summary Significant progress in clinical translation of AAV-mediated cardiac gene therapy has been achieved in recent years. This will prompt further clinical trials, and positive results could open a new era for cardiac gene therapy.

Original languageEnglish
Pages (from-to)228-234
Number of pages7
JournalCurrent Opinion in Cardiology
Issue number3
StatePublished - 27 May 2015


  • adeno-associated virus
  • arrhythmias
  • gene therapy
  • heart failure
  • sarcoplasmic reticulum calcium ATPase pump


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