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

Molecular Neurodegeneration

Xiao-Jiang Li¹  Xiangyu Guo²  Sen Yan²  Weili Yang²  Zhuchi Tu² 

[1] Department of Human Genetics, Emory University School of Medicine, Atlanta 30322, GA, USA;Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing 100101, China

关键词: Animal model;    Neurodegenerative diseases;    Non-human primates;    CRISPR/Cas9;   

Others  :  1221856
DOI  :  10.1186/s13024-015-0031-x

 received in 2015-06-08, accepted in 2015-07-24,  发布年份 2015

【 摘 要 】

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.

【 授权许可】

   
2015 Tu et al.

【 图 表 】

Fig. 1.

Fig. 2.

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