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心脏病的基因治疗:分子靶点、载体及心肌递送方式

Gene therapy for heart disease: molecular targets, vectors and modes of delivery to myocardium.

作者信息

Scimia Maria Cecilia, Cannavo Alessandro, Koch Walter J

机构信息

Department of Pharmacology, Center for Translational Medicine, Temple University School of Medicine, 3500 N Broad St, MERB 941, Philadelphia, PA 19140, USA.

出版信息

Expert Rev Cardiovasc Ther. 2013 Aug;11(8):999-1013. doi: 10.1586/14779072.2013.818813.

Abstract

Despite the numerous hurdles that gene therapy has encountered along the way, clinical trials over the last few years are showing promising results in many fields of medicine, including cardiology, where many targets are moving toward clinical development. In this review, the authors discuss the current state of the art in terms of clinical and preclinical development. They also examine vector technology and available vector-delivery strategies.

摘要

尽管基因治疗在发展过程中遇到了众多障碍,但过去几年的临床试验在包括心脏病学在内的许多医学领域都显示出了有前景的结果,在心脏病学领域,许多靶点正朝着临床开发迈进。在这篇综述中,作者讨论了临床和临床前开发方面的当前技术水平。他们还研究了载体技术和可用的载体递送策略。

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