Current Heart Failure Reports

, Volume 15, Issue 6, pp 340–349 | Cite as

Gene Therapy for Heart Failure: New Perspectives

  • Khatia Gabisonia
  • Fabio A. RecchiaEmail author
Pathophysiology: Neuroendocrine, Vascular, and Metabolic Factors (S. Katz, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Pathophysiology: Neuroendocrine, Vascular, and Metabolic Factors


Purpose of Review

The current knowledge of pathophysiological and molecular mechanisms responsible for the genesis and development of heart failure (HF) is absolutely vast. Nonetheless, the hiatus between experimental findings and therapeutic options remains too deep, while the available pharmacological treatments are mostly seasoned and display limited efficacy. The necessity to identify new, non-pharmacological strategies to target molecular alterations led investigators, already many years ago, to propose gene therapy for HF. Here, we will review some of the strategies proposed over the past years to target major pathogenic mechanisms/factors responsible for severe cardiac injury developing into HF and will provide arguments in favor of the necessity to keep alive research on this topic.

Recent Findings

After decades of preclinical research and phases of enthusiasm and disappointment, clinical trials were finally launched in recent years. The first one to reach phase II and testing gene delivery of sarcoendoplasmic reticulum calcium ATPase did not yield encouraging results; however, other trials are ongoing, more efficient viral vectors are being developed, and promising new potential targets have been identified. For instance, recent research is focused on gene repair, in vivo, to treat heritable forms of HF, while strong experimental evidence indicates that specific microRNAs can be delivered to post-ischemic hearts to induce regeneration, a result that was previously thought possible only by using stem cell therapy.


Gene therapy for HF is aging, but exciting perspectives are still very open.


Heart failure Gene therapy Duchenne cardiomyopathy Hippo pathway AAV miRNA 


Compliance with Ethical Standards

Conflict of Interest

The authors declare that they have no conflict of interest.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


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© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Institute of Life Sciences, Fondazione Toscana Gabriele MonasterioScuola Superiore Sant’AnnaPisaItaly
  2. 2.Cardiovascular Research Center, Lewis Katz School of MedicineTemple UniversityPhiladelphiaUSA

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