Knock in of a hexanucleotide repeat expansion in the C9orf72 gene induces ALS in rats.
Wei DongLi ZhangCaixian SunXiang GaoFeifei GuanJing LiWei ChenYuanwu MaLianfeng ZhangPublished in: Animal models and experimental medicine (2020)
Motor defects in KI rats may result from neurotoxicity caused by HRE and the resulting reduction in C9orf72 protein due to haploinsufficiency. These KI rats could be a useful model for investigating the contributions of loss-of-function to neurotoxicity in C9orf72-related ALS.