针对心血管疾病的特定位点基因疗法。

Ilia Fishbein, Michael Chorny, Robert J Levy
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引用次数: 0

摘要

基因疗法在治疗心血管疾病方面大有可为,可为遗传性疾病和后天性病理生理(如动脉硬化、心力衰竭和心律失常)提供新的治疗方案。DNA 重组技术和人类基因组测序为心血管疾病提供了大量候选治疗基因。然而,心血管疾病基因治疗领域的进展不大;进展有限的主要原因之一是缺乏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。本综述总结了在心血管基因治疗的特异性位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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Site-specific gene therapy for cardiovascular disease.

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.

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