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Short interfering RNA (siRNA), a novel therapeutic tool acting on angiogenesis

机译:短干扰RNA(siRNA),一种作用于血管生成的新型治疗工具

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摘要

The formation of new blood vessels, uncontrolled cell expansions and invasions are the common feature of cancer, neovascular inflammatory and ocular diseases, such as age-related macular degeneration (AMD). Short interfering RNA (siRNA) and short-hairpin RNA (shRNA) have recently helped extend our understanding of the mechanisms regulating angiogenesis and tumor developments. Moreover, the early success of these tools has reinforced the therapeutic hopes of preventing endogenous or exogenous gene translation. In vivo experiments using several animal tumor models and human pre-clinical trials augured many benefits to control protein expression and cell signaling. The high specificity of siRNA and shRNA to target a protein is crucial to design a new generation of therapeutic agents. At the present, several investigations are focused on the understanding of both gene function and the proof-of-concept for siRNA-mediated anti-angiogenesis. Taken together, in vitro and in vivo studies shed light on the efficiency of siRNA as a new alternative therapeutic agent.
机译:新血管的形成,不受控制的细胞扩增和侵袭是癌症,新血管炎性和眼部疾病(例如与年龄有关的黄斑变性(AMD))的共同特征。短干扰RNA(siRNA)和短发夹RNA(shRNA)最近帮助扩展了我们对调节血管生成和肿瘤发展的机制的了解。而且,这些工具的早期成功增强了预防内源或外源基因翻译的治疗希望。使用几种动物肿瘤模型的体内实验和人类临床前试验预示了控制蛋白质表达和细胞信号传导的许多益处。 siRNA和shRNA靶向蛋白质的高特异性对于设计新一代治疗剂至关重要。目前,一些研究集中在对基因功能的理解和siRNA介导的抗血管生成的概念验证上。总之,体外和体内研究揭示了siRNA作为新型替代治疗剂的效率。

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