Preclinical models: needed in translation? A Pro/Con debate.
Preclinical models: needed in translation? A Pro/Con debate.
复制标题
临床前模型：翻译需要吗？
DOI：
10.1002/mds.26010
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发表时间：
2014
期刊：
影响因子：
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通讯作者：
Pouladi,MahmoudA
中科院分区：
文献类型：
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作者：
Philips,Thomas;Rothstein,JeffreyD;Pouladi,MahmoudA
神经退行性疾病如肌萎缩性侧索硬化症、阿尔茨海默氏病、帕金森氏病和亨廷顿病（HD）的致病突变和许多诱发风险因素的发现导致了大量疾病的遗传动物模型的开发。例如，在HD的情况下，已经产生了超过20种不同的转基因啮齿动物模型。这些模型在为疾病机制提供新的见解方面具有巨大的价值，有望加速开发可以延迟疾病发作或减缓疾病进展的疗法。然而，尽管广泛使用这些模型，但尚未开发出有效的HD治疗方法。在这里，我们讨论了动物模型的价值，突出了它们在HD转化研究中的优势和不足。国际帕金森和运动障碍协会（International Parkinson and Movement Disorder Society）
The discovery of the causative mutations and many of the predisposing risk factors for neurodegenerative disorders such as Amyotrophic Lateral Sclerosis, Alzheimer's, Parkinson's, and Huntington's disease (HD), has led to the development of a large number of genetic animal models of disease. In the case of HD, for example, over 20 different transgenic rodent models have been generated. These models have been of immense value in providing novel insights into mechanisms of disease, with the promise of accelerating the development of therapies that can delay the onset or slow the progression of the disease. Yet, despite extensive use of such models, no effective treatment for HD has been developed. Here, we discuss the value of animal models, highlighting their strengths and shortcomings in the context of translational research for HD. © 2014 International Parkinson and Movement Disorder Society