Adeno-Associated Virus Vector Delivery to the Heart

  • Lawrence T. Bish
  • H. Lee SweeneyEmail author
  • Oliver J. Müller
  • Raffi Bekeredjian
Part of the Methods in Molecular Biology book series (MIMB, volume 807)


Cardiac gene transfer may serve as a novel therapeutic approach in the treatment of heart disease. For it to reach its full potential, methods for highly efficient cardiac gene transfer must be available to investigators so that informative preclinical data can be collected and evaluated. We have recently optimized AAV-mediated cardiac gene transfer protocols in both the mouse and rat. In the mouse, we have developed a procedure for intrapericardial delivery of vector in the neonate and successfully applied intravenous injections in adult animals. In the rat, we have developed a procedure for direct injection of vector into the myocardium in adults and established a protocol for vector delivery into the left ventricular anterior wall by ultrasound-targeted destruction of microbubbles loaded with AAV. Each protocol can be used to achieve safe and efficient cardiac gene transfer in the model of choice.

Key words

Heart Gene therapy Adeno-associated virus AAV Animal model Microbubbles Ultrasound 



This work was supported by a grant from the NHLBI (P01-HL059407 to HLS) and by TG-HL-007748 to LTB. Further support was obtained by a grant of the Deutsche Forschungsgemeinschaft (MU 1654/3-2) and the Bundesministerium für Bildung und Forschung (01GU0527) to OJM as well as by the BioFuture grant of the Bundesministerium für Bildung und Forschung to RB. We thank Stefanie Schinkel for her help in preparing this manuscript.


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Copyright information

© Springer Science+Business Media, LLC 2012

Authors and Affiliations

  • Lawrence T. Bish
    • 1
  • H. Lee Sweeney
    • 1
    Email author
  • Oliver J. Müller
    • 2
  • Raffi Bekeredjian
    • 2
  1. 1.Department of PhysiologyUniversity of Pennsylvania School of MedicinePhiladelphiaUSA
  2. 2.Internal MedicineUniversity of HeidelbergHeidelbergGermany

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