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Gene Therapy for Heart Failure: New Perspectives

  • Pathophysiology: Neuroendocrine, Vascular, and Metabolic Factors (S. Katz, Section Editor)
  • Published:
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Abstract

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.

Summary

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

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  1. Institute of Life Sciences, Fondazione Toscana Gabriele Monasterio, Scuola Superiore Sant’Anna, Piazza Martiri della Liberta` 33, 56127, Pisa, Italy

    Khatia Gabisonia & Fabio A. Recchia

  2. Cardiovascular Research Center, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, USA

    Fabio A. Recchia

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Gabisonia, K., Recchia, F.A. Gene Therapy for Heart Failure: New Perspectives. Curr Heart Fail Rep 15, 340–349 (2018). https://doi.org/10.1007/s11897-018-0410-z

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