Current Methods in Cardiac Gene Therapy: Overview

Methods Mol Biol. 2017:1521:3-14. doi: 10.1007/978-1-4939-6588-5_1.

Abstract

During the last decade, there has been a significant progress toward clinical translation in the field of cardiac gene therapy based on extensive preclinical data. However, despite encouraging positive results in early phase clinical trials, more recent larger trials reported only neutral results. Nevertheless, the field has gained important knowledge from these trials and is leading to the development of more cardiotropic vectors and improved delivery systems. It has become more evident that humans are more resistant to therapeutic transgene expression compared to experimental animals and thus refinement in gene delivery tools and methods are essential for future success. We provide an overview of the current status of cardiac gene therapy focusing on gene delivery tools and methods. Newer technologies, devices, and approaches will undoubtedly lead to more promising clinical results in the near future.

Keywords: Adeno-associated vectors; Cardiac gene therapy; Cardiotropic; Gene delivery; Heart failure; Percutaneous delivery; Promoters; Surgical delivery.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors / metabolism
  • Heart / physiology*
  • Humans
  • Promoter Regions, Genetic / genetics