S100A1 gene therapy for heart failure: a novel strategy on the verge of clinical trials.

S100A1 gene therapy for heart failure: a novel strategy on the verge of clinical trials.
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S100A1 基因治疗心力衰竭:一种即将进入临床试验的新策略。

DOI:
10.1016/j.yjmcc.2010.08.012
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发表时间:
2011
影响因子:
5
通讯作者:
Most,Patrick
Most,Patrick
中科院分区:
医学2区
文献类型:
--
作者:
Rohde,David;Brinks,Henriette;Ritterhoff,Julia;Qui,Gang;Ren,Shumei;Most,Patrick

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心力衰竭(HF)是各种心血管疾病的共同终点,其患病率急剧增加。由于目前可用的治疗策略不能终止疾病的进展,HF仍然与不良的临床预后相关。在潜在的分子机制中,心肌细胞Ca 2+循环完整性的丧失在疾病的病理生理发展和进展中起关键作用。心肌细胞EF-手Ca 2+传感器蛋白S100 A1作为肌浆网(SR),肌节和线粒体功能的调节剂出现,在心脏生理和功能障碍中起着重要作用。在这篇综述中,我们的目标是概括翻译的S100 A1为基础的调查从第一次临床观察基础研究实验回到一个接近临床的设置今天的临床试验的边缘。我们还讨论了进一步发展“第二代”基因治疗的需求,并讨论了S100 A1基因治疗心力衰竭的治疗潜力,作为未来心脏病学家有前途的新策略。
Representing the common endpoint of various cardiovascular disorders, heart failure (HF) shows a dramatically growing prevalence. As currently available therapeutic strategies are not capable of terminating the progress of the disease, HF is still associated with a poor clinical prognosis. Among the underlying molecular mechanisms, the loss of cardiomyocyte Ca2+cycling integrity plays a key role in the pathophysiological development and progression of the disease. The cardiomyocyte EF-hand Ca2+sensor protein S100A1 emerged as a regulator both of sarcoplasmic reticulum (SR), sarcomere and mitochondrial function implicating a significant role in cardiac physiology and dysfunction. In this review, we aim to recapitulate the translation of S100A1-based investigation from first clinical observations over basic research experiments back to a near-clinical setting on the verge of clinical trials today. We also address needs for further developments towards “second-generation” gene therapy and discuss the therapeutic potential of S100A1 gene therapy for HF as a promising novel strategy for future cardiologists.
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发表时间: 2005-12-01
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