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Potential of gene therapy as a treatment for heart failure
Roger J. Hajjar
Roger J. Hajjar
Published January 2, 2013
Citation Information: J Clin Invest. 2013;123(1):53-61. https://doi.org/10.1172/JCI62837.
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Potential of gene therapy as a treatment for heart failure

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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.

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Roger J. Hajjar

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