Adeno-Associated Virus Vector Delivery to the Heart
Adeno-Associated Virus Vector Delivery to the Heart
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DOI：
10.1007/978-1-61779-370-7_9
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发表时间：
2011-01-01
期刊：
影响因子：
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通讯作者：
Bekeredjian, Raffi
中科院分区：
文献类型：
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作者：
Bish, Lawrence T.;Sweeney, H. Lee;Bekeredjian, Raffi
心脏基因转移可能成为治疗心脏病的一种新的治疗方法。为了充分发挥其潜力，研究人员必须能够获得高效的心脏基因转移方法，以便收集和评估临床前的信息数据。我们最近在小鼠和大鼠身上优化了AAV介导的心脏基因转移方案。在小鼠中，我们已经开发了一种新生儿心包内注射载体的程序，并成功地在成年动物身上应用了静脉注射。在大鼠中，我们已经开发了一种将载体直接注射到成年大鼠心肌中的程序，并建立了一种通过超声靶向破坏携带AAV的微泡将载体运送到左室前壁的方案。每种方案都可以用来实现安全高效的心脏基因转移模式的选择。
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.