Potential of gene therapy as a treatment for heart failure

Roger J. Hajjar

Research output: Contribution to journalReview articlepeer-review

72 Scopus citations

Abstract

Advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology, make gene-based therapy a promising treatment option for heart conditions. Cardiovascular gene therapy has benefitted from recent advancements in vector technology, design, and delivery modalities. There is a critical need to explore new therapeutic approaches in heart failure, and gene therapy has emerged as a viable alternative. 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. The recent successful and safe completion of a phase 2 trial targeting the cardiac sarcoplasmic/endoplasmic reticulum Ca2+ ATPase pump (SERCA2a) has the potential to open a new era for gene therapy for heart failure.

Original languageEnglish
Pages (from-to)53-61
Number of pages9
JournalJournal of Clinical Investigation
Volume123
Issue number1
DOIs
StatePublished - 2 Jan 2013

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