Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice
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A. Krainer | Yidi Sun | Min-ting Lin | Ning Wang | Erwei Zuo | Wenqin Ying | Hui Yang | Xin-Xin Guo | Xinde Hu | Xiaowen Shen | Qifang Wang | Linyu Shi | Qi-Jie Zhang | Changyang Zhou | Cheng Tang | Xiang Lin | Jin-Jing Li | Shuang Wu | Ying-qian Lu | Lu-Lu Lai | Li-xiang Ma | Haizhu Chen | He Li | Wan-Jin Chen | Erwei Zuo