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Disruption of Splicing-Regulatory Elements Using CRISPR/Cas9 to Rescue Spinal Muscular Atrophy in Human Ipscs and Mice

Jin-Jing Li,Xiang Lin,Cheng Tang,Ying-Qian Lu,Xinde Hu,Erwei Zuo,He Li,Wenqin Ying,Yidi Sun,Lu-Lu Lai,Hai-Zhu Chen,Xin-Xin Guo,Qi-Jie Zhang,Shuang Wu,Changyang Zhou,Xiaowen Shen,Qifang Wang,Min-Ting Lin,Li-Xiang Ma,Ning Wang,Adrian R. Krainer,Linyu Shi,Hui Yang,Wan-Jin Chen

NATIONAL SCIENCE REVIEW（2020）

Cited 24|Views42
Key words
spinal muscular atrophy,SMN2,splicing-regulatory elements,CRISPR/Cas9,germline correction
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