S100A1 gene therapy in small and large animals.
S100A1 gene therapy in small and large animals.
复制标题
S100A1 基因治疗小型和大型动物。
DOI：
10.1007/978-1-62703-230-8_25
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发表时间：
2013
期刊：
影响因子：
--
通讯作者：
Pleger,SvenT
中科院分区：
文献类型：
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作者：
Most,Patrick;Raake,Philip;Weber,Christophe;Katus,HugoA;Pleger,SvenT
在健康动物和各种心血管疾病模型中，心肌内基因传递是研究目标蛋白与心脏收缩功能、肥大和能量状态的相关性的一种有价值的技术。啮齿动物模型用于筛选效应和研究分子机制，而更能反映人体解剖学、生理学和功能的大型动物模型是转化性治疗方法的必然选择。由非心脏选择性启动子或心脏选择性启动子驱动其表达的目的基因被克隆到病毒载体中。然后，这种载体使用适当的给药途径来靶向心脏并在心肌中实现高效的蛋白质表达。在这里，我们描述了在小动物和大动物的缺血后心力衰竭模型中的心肌基因治疗，用于揭示小钙传感器蛋白S100A1的正性肌力抑制、抗肥厚和促能量作用。
Myocardial in vivo gene delivery is a valuable technique to investigate the relevance of a protein of interest on cardiac contractile function, hypertrophy, and energy state in healthy animals as well as in a variety of models of cardiovascular disease. Rodent models are used to screen effects and to investigate molecular mechanisms, while large animal models, more closely reflecting human anatomy, physiology, and function, are inevitable for translational therapeutic approaches. The gene of interest, whose expression is driven by a non-cardioselective or cardioselective promotor is cloned into a viral vector. This vehicle is then delivered using an appropriate administration route to target the heart and to achieve efficient protein expression in myocardium.Here we describe myocardial gene therapy in small and large animal models of postischemic heart failure used to reveal the positive inotrope, antihypertrophic, and pro-energetic action of the small calcium sensor protein S100A1.