Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome
利用STEP-RNP进行全脑基因组编辑治疗安格曼综合征
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doi:10.1101/2025.11.13.684643
Lu, Xiaona; Xie, Ying; Bao, Youmei; Yu, Jiang; Wang, Zefeng; Fan, Jiali; Sheu, Wendy C; Long, Gretchen; Tu, Zewei; Tong, Jie; Manquen, Garret; Li, Yichao; Katta, Varun; Chow, Ryan; Chen, Binfan; Meng, Zhouqi; Ma, Ying-Hong; Qiu, Caihong; Chen, Sidi; Tsai, Shengdar Q; De Kumar, Bony; Hendry, Caroline E; Jiang, Yong-Hui; Zhou, Jiangbing