title: CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases.
abstract: 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.
authors.affiliation: Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing, 100101, China.
authors.forename: Zhuchi
authors.initials: Z
authors.lastname: Tu
authors.affiliation: Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing, 100101, China.
authors.forename: Weili
authors.initials: W
authors.lastname: Yang
authors.affiliation: Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing, 100101, China.
authors.forename: Sen
authors.initials: S
authors.lastname: Yan
authors.affiliation: Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing, 100101, China. guosapphire@yahoo.com.
authors.forename: Xiangyu
authors.initials: X
authors.lastname: Guo
authors.affiliation: Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Beijing, 100101, China. xli2@emory.edu.|Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, 30322, USA. xli2@emory.edu.
authors.forename: Xiao-Jiang
authors.initials: XJ
authors.lastname: Li
mesh_terms: D000818:Animals; D030801:Animals, Genetically Modified; D001426:Bacterial Proteins / Q000378:metabolism; D064130:CRISPR-Associated Proteins / Q000378:metabolism; D064113:CRISPR-Cas Systems / Q000235:genetics*; D059766:DNA End-Joining Repair; D004195:Disease Models, Animal; D004624:Embryo Transfer; D004720:Endonucleases / Q000378:metabolism; D005260:Female; D005818:Genetic Engineering / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008297:Male; D009030:Mosaicism; D016296:Mutagenesis; D019636:Neurodegenerative Diseases / Q000235:genetics*; D011323:Primates / Q000235:genetics*; D000094704:RNA, Guide, CRISPR-Cas Systems / Q000235:genetics*; D011993:Recombinant Fusion Proteins / Q000378:metabolism; D012756:Sheep / Q000235:genetics*; D013045:Species Specificity; D020554:Sperm Injections, Intracytoplasmic; D013297:Streptococcus pyogenes / Q000201:enzymology; D034421:Sus scrofa / Q000235:genetics*
chemical_list: D001426:Bacterial Proteins; D064130:CRISPR-Associated Proteins; D000094704:RNA, Guide, CRISPR-Cas Systems; D011993:Recombinant Fusion Proteins; D004720:Endonucleases