Precise excision of expanded GGC repeats in NOTCH2NLC via CRISPR/Cas9 for treating neuronal intranuclear inclusion disease.
利用 CRISPR/Cas9 技术精确切除 NOTCH2NLC 中扩增的 GGC 重复序列,用于治疗神经元核内包涵体病。
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-026-68385-5
Xie Nina, Pan Yongcheng, Tong Huichun, Lin Yingqi, Jiang Ying, Wang Zhiqin, Wan Juan, Zhang Wendiao, Wang Xinhui, Sun Xiaobo, Yan Sen, Yin Peng, Sun Qiying, Qi Chengzhi, Tian Yun, Shen Lu, Jiang Hong, Liang Desheng, Tang Beisha, Li Shihua, Li Xiao-Jiang, Liu Qiong