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AAV vectors for efficient gene delivery to rodent hearts

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

7 Scopus citations

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.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages311-332
Number of pages22
DOIs
StatePublished - 2019

Publication series

NameMethods in Molecular Biology
Volume1950
ISSN (Print)1064-3745

Keywords

  • AAV characterization
  • AAV delivery
  • AAV production
  • AAV9
  • Adeno-associated virus
  • Mouse
  • Rat

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