Cardiac gene therapy: from concept to reality.
Cardiac gene therapy: from concept to reality.
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DOI：
10.1007/s11897-011-0077-1
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发表时间：
2012-03
影响因子：
--
通讯作者：
Hajjar, Roger J
中科院分区：
文献类型：
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作者：
Kratlian, Razmig Garo;Hajjar, Roger J
心力衰竭的发病率在世界范围内呈上升趋势，尤其是在工业化国家。虽然目前的治疗方式已经成功地稳定了心力衰竭的进程，但发病率和死亡率仍然很高，仍然非常需要创新突破，为晚期心力衰竭患者提供新的治疗策略。过去几年，人们对心脏衰竭的分子基础有了更深入的了解，为调节细胞环境的新策略铺平了道路。因此，基因治疗最近成为一种强有力的工具，为减轻心力衰竭提供了一种新的范例。目前心力衰竭的基因治疗研究主要集中在探索潜在的细胞靶点，临床前和临床研究正在朝着这一目标的实现进行。努力还包括开发复杂的病毒载体和载体传递方法，以有效地转导心肌细胞。
Heart failure is increasing in incidence throughout the world, especially in industrialized countries. Although the current therapeutic modalities have been successful in stabilizing the course of heart failure, morbidity and mortality remain quite high and there remains a great need for innovative breakthroughs that will offer new treatment strategies for patients with advanced forms of the disease. The past few years have witnessed a greater understanding of the molecular underpinnings of the failing heart, paving the way for novel strategies in modulating the cellular environment. As such, gene therapy has recently emerged as a powerful tool offering the promise of a new paradigm for alleviating heart failure. Current gene therapy research for heart failure is focused on exploring potential cellular targets and preclinical and clinical studies are ongoing toward the realization of this goal. Efforts also include the development of sophisticated viral vectors and vector delivery methods for efficient transduction of cardiomyocytes.