Protocol

Adeno-Associated Virus

Volume 807 of the series Methods in Molecular Biology pp 219-237

Date:

Adeno-Associated Virus Vector Delivery to the Heart

  • Lawrence T. BishAffiliated withDepartment of Physiology, University of Pennsylvania School of Medicine
  • , H. Lee SweeneyAffiliated withDepartment of Physiology, University of Pennsylvania School of Medicine Email author 
  • , Oliver J. MüllerAffiliated withInternal Medicine, University of Heidelberg
  • , Raffi BekeredjianAffiliated withInternal Medicine, University of Heidelberg

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Abstract

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