Use of Adeno-Associated Virus Vector for Cardiac Gene Delivery in Large-Animal Surgical Models of Heart Failure

Michael G. Katz, Anthony S. Fargnoli, Thomas Weber, Roger J. Hajjar, Charles R. Bridges

Research output: Contribution to journalArticlepeer-review

22 Scopus citations

Abstract

The advancement of gene therapy-based approaches to treat heart disease represents a need for clinically relevant animal models with characteristics equivalent to human pathologies. Rodent models of cardiac disease do not precisely reproduce heart failure phenotype and molecular defects. This has motivated researchers to use large animals whose heart size and physiological processes more similar and comparable to those of humans. Today, adeno-associated viruses (AAV)-based vectors are undoubtedly among the most promising DNA delivery vehicles. Here, AAV biology and technology are reviewed and discussed in the context of their use and efficacy for cardiac gene delivery in large-animal models of heart failure, using different surgical approaches. The remaining challenges and opportunities for the use of AAV-based vector delivery for gene therapy applications in the clinic are also highlighted.

Original languageEnglish
Pages (from-to)157-164
Number of pages8
JournalHuman Gene Therapy Clinical Development
Volume28
Issue number3
DOIs
StatePublished - Sep 2017

Keywords

  • AAV virus
  • animal models of heart failure
  • cardiac gene delivery
  • heart disease

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