The benefits and limitations of animal models for translational research in neurodegenerative diseases
The benefits and limitations of animal models for translational research in neurodegenerative diseases
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DOI：
10.1038/nm.2224
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发表时间：
2010-11-01
期刊：
影响因子：
82.9
通讯作者：
Jucker, Mathias
中科院分区：
文献类型：
--
作者：
Jucker, Mathias
与年龄相关的神经退行性疾病在很大程度上仅限于人类，很少在动物身上自发发生。基因工程小鼠模型概括了相应人类疾病的各个方面，并有助于研究疾病机制和测试治疗策略。如果在其有效性范围内考虑，小鼠模型可以预测临床结果。翻译失败与其说是模型的不完整性质造成的，不如说是不充分的临床前研究和对模型的误解造成的。这篇评论总结了当前的模型，并强调了我们应该对动物模型提出的关键问题，以及用当前模型无法回答的问题。
Age-related neurodegenerative diseases are largely limited to humans and rarely occur spontaneously in animals. Genetically engineered mouse models recapitulate aspects of the corresponding human diseases and are instrumental in studying disease mechanisms and testing therapeutic strategies. If considered within the range of their validity, mouse models have been predictive of clinical outcome. Translational failure is less the result of the incomplete nature of the models than of inadequate preclinical studies and misinterpretation of the models. This commentary summarizes current models and highlights key questions we should be asking about animal models, as well as questions that cannot be answered with the current models.