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AAV Vectors for Efficient Gene Delivery to Rodent Hearts

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Adeno-Associated Virus Vectors

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1950))

Abstract

Currently, gene therapy is one of the most promising fields in biomedicine, with great therapeutic potential for an array of inherited and acquired diseases. Adeno-associated viral (AAV) vectors have emerged as promising tools to deliver selectively a therapeutic payload to target organs, including the heart. In this chapter, we describe the production and quality control of recombinant AAV (rAAV) vectors of the serotype 9, the most cardiotropic AAV serotype when delivered systemically in rodents. We also describe the systemic administration of rAAV vectors and the local delivery of rAAV vectors by direct intramyocardial injection. Taken together, the methods described in this chapter will allow the reader to deliver efficiently therapeutic genes to the rodent heart, both globally and regionally.

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Acknowledgments

We would like to thank Elena Chepurko, Malik Bisserier, and Anthony Fargnoli for helpful discussions. This work is supported by NIH P50 HL112324, R01 HL119046, R01 HL117505, R01 HL128099, R01 HL129814, R01 HL131404 and Trans-Atlantic Network of Excellence grants 13CVD01 and 14CVD03 from the Leducq Foundation.

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Authors and Affiliations

  1. Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York City, NY, USA

    Estrella Lopez-Gordo, Erik Kohlbrenner, Michael G. Katz & Thomas Weber

  2. Graduate School of Biomedical Sciences, Icahn School of Medicine at Mount Sinai, New York City, NY, USA

    Thomas Weber

Authors
  1. Estrella Lopez-Gordo
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  2. Erik Kohlbrenner
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  3. Michael G. Katz
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  4. Thomas Weber
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Corresponding author

Correspondence to Thomas Weber .

Editor information

Editors and Affiliations

  1. Department of Neurosciences, University of California, San Diego, La Jolla, CA, USA

    Michael J. Castle

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© 2019 Springer Science+Business Media, LLC, part of Springer Nature

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Lopez-Gordo, E., Kohlbrenner, E., Katz, M.G., Weber, T. (2019). AAV Vectors for Efficient Gene Delivery to Rodent Hearts. In: Castle, M. (eds) Adeno-Associated Virus Vectors. Methods in Molecular Biology, vol 1950. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9139-6_19

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  • DOI: https://doi.org/10.1007/978-1-4939-9139-6_19

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-9138-9

  • Online ISBN: 978-1-4939-9139-6

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