Animal models are extremely valuable to help us understand the pathogenesis of neurodegenerative disorders and to find treatments for them. Since large animals are more like humans than rodents, they make good models to identify the important pathological events that may be seen in humans but not in small animals; large animals are also very important for validating effective treatments or confirming therapeutic targets. Due to the lack of embryonic stem cell lines from large animals, it has been difficult to use traditional gene targeting technology to establish large animal models of neurodegenerative diseases. Recently, CRISPR/Cas9 was used successfully to genetically modify genomes in various species. Here we discuss the use of CRISPR/Cas9 technology to establish large animal models that can more faithfully mimic human neurodegenerative diseases.

中文翻译：

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CRISPR/Cas9：用于建立神经退行性疾病大型动物模型的强大基因工程工具。


动物模型对于帮助我们了解神经退行性疾病的发病机制并找到治疗方法非常有价值。由于大型动物比啮齿类动物更像人类，因此它们可以作为很好的模型来识别可能在人类中看到但在小动物中不可见的重要病理事件；大型动物对于验证有效治疗或确认治疗靶点也非常重要。由于缺乏大型动物的胚胎干细胞系，利用传统的基因打靶技术建立大型动物神经退行性疾病模型一直存在困难。最近，CRISPR/Cas9已成功用于对多个物种的基因组进行遗传修饰。在这里我们讨论利用CRISPR/Cas9技术建立能够更忠实地模拟人类神经退行性疾病的大型动物模型。