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心脏基因治疗中的基因传递方法。

Gene delivery methods in cardiac gene therapy.

机构信息

Cardiovascular Research Center, Mount Sinai School of Medicine, New York, NY 10029-6574, USA.

出版信息

J Gene Med. 2011 Oct;13(10):566-72. doi: 10.1002/jgm.1609.

Abstract

Gene therapy for the treatment of heart failure is emerging as a multidisciplinary field demonstrating advances with respect to identifying key signaling pathways, modernized vector creation and delivery technologies. Although these discoveries offer significant progress, selecting optimal methods for the vector delivery remains a key component for efficient cardiac gene therapy to validate the targets in rodent models and to test clinically relevant ones in pre-clinical models. Although the goals of higher transduction efficiency and cardiac specificity can be achieved with several delivery methods, the invasiveness and patient safety remain unclear for clinical application. In this review, we discuss various features of the currently available vector delivery methods for cardiac gene therapy.

摘要

基因治疗心力衰竭作为一个多学科领域正在兴起,在确定关键信号通路、现代化载体创建和传递技术方面取得了进展。尽管这些发现取得了重大进展,但选择载体传递的最佳方法仍然是心脏基因治疗的关键组成部分,以验证啮齿动物模型中的靶点,并在临床前模型中测试临床相关的靶点。虽然几种传递方法可以实现更高的转导效率和心脏特异性,但对于临床应用,其侵入性和患者安全性仍不清楚。在这篇综述中,我们讨论了目前用于心脏基因治疗的各种载体传递方法的特点。

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