Gene therapy for heart failure

Mario T. Njeim, Roger J. Hajjar

Research output: Contribution to journalReview articlepeer-review

15 Scopus citations


Despite the progress achieved in conventional treatment modalities, heart failure remains a major cause of mortality and morbidity. The identification of novel signaling pathways has provided a solid scientific rationale which has stimulated preclinical development of gene-based therapies for heart failure. Advances in somatic gene transfer technologies have been crucial to the advent of the first human clinical trials which are currently in progress. As these and other trials of gene transfer-based therapies are initiated, these approaches have generated excitement and hope for novel treatments for cardiovascular disease. In this review, we present a summary of advancements in construction of different vectors and methods of delivery that have been used for specific myocardial gene delivery. In addition, we will show results from studies focusing on the use of gene therapy to target heart failure mechanisms in animal models of cardiac dysfunction. Finally, we discuss the limited but highly promising results from clinical studies that have served as catalysts to translate preclinical achievements towards new treatment modalities for heart failure.

Original languageEnglish
Pages (from-to)477-485
Number of pages9
JournalArchives of Cardiovascular Diseases
Issue number8-9
StatePublished - Aug 2010


  • Gene therapy
  • Heart failure


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