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心血管疾病的位点特异性基因治疗。

Site-specific gene therapy for cardiovascular disease.

作者信息

Fishbein Ilia, Chorny Michael, Levy Robert J

机构信息

The Children's Hospital of Philadelphia, Abramson Research Center, Philadelphia, PA 19104, USA.

出版信息

Curr Opin Drug Discov Devel. 2010 Mar;13(2):203-13.

Abstract

Gene therapy holds considerable promise for the treatment of cardiovascular disease and may provide novel therapeutic solutions for both genetic disorders and acquired pathophysiologies such as arteriosclerosis, heart failure and arrhythmias. Recombinant DNA technology and the sequencing of the human genome have made a plethora of candidate therapeutic genes available for cardiovascular diseases. However, progress in the field of gene therapy for cardiovascular disease has been modest; one of the key reasons for this limited progress is the lack of gene delivery systems for localizing gene therapy to specific sites to optimize transgene expression and efficacy. This review summarizes progress made toward the site-specific delivery of cardiovascular gene therapy and highlights selected promising novel approaches.

摘要

基因治疗在心血管疾病的治疗方面具有巨大潜力,并且可能为遗传性疾病以及诸如动脉硬化、心力衰竭和心律失常等后天性病理生理状况提供新的治疗方案。重组DNA技术和人类基因组测序已催生出大量可用于治疗心血管疾病的候选治疗基因。然而,心血管疾病基因治疗领域的进展较为有限;取得这一有限进展的关键原因之一是缺乏能够将基因治疗定位到特定部位以优化转基因表达和疗效的基因递送系统。本综述总结了心血管疾病基因治疗在定点递送方面取得的进展,并重点介绍了一些有前景的新方法。

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