Nature communications
Precise excision of expanded GGC repeats in NOTCH2NLC via CRISPR/Cas9 for treating neuronal intranuclear inclusion disease.
Nina Xie, Yongcheng Pan, Huichun Tong, Yingqi Lin, Ying Jiang, Zhiqin Wang, Juan Wan, Wendiao Zhang, Xinhui Wang, Xiaobo Sun, Sen Yan, Peng Yin, Qiying Sun, Chengzhi Qi, Yun Tian
Published: 202610.1038/s41467-026-68385-5
Abstract
Neuronal intranuclear inclusion disease (NIID) is an adult-onset neurodegenerative disease caused by expanded GGC repeats in the 5' untranslated region of the human-specific NOTCH2NLC gene. The high sequence similarity between NOTCH2NLC and its paral…
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