CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases

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• 作者：Zhuchi Tu ; Weili Yang ; Sen Yan ; Xiangyu Guo ; Xiao-Jiang Li
• 关键词：CRISPR/Cas9 ; Non ; human primates ; Neurodegenerative diseases ; Animal model
• 刊名：Molecular Neurodegeneration
• 出版年：2015
• 出版时间：December 2015
• 年：2015
• 卷：10
• 期：1
• 全文大小：849 KB
• 刊物主题：Neurosciences; Neurology; Molecular Medicine;
• 出版者：BioMed Central
• ISSN：1750-1326

文摘

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.