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[期刊论文] 作者:Lu-Lu Lai,Hai-Zhu Chen,Xin-Xin Guo,Qi-Jie Zhang,Shuang Wu,Changyang Zhou,Xiaowen Shen,Qifang Wang,Min-Ting L, 来源:国家科学评论(英文版) 年份:2020
We here report a genome-editing strategy to correct spinal muscular atrophy (SMA).Rather than directly targeting the pathogenic exonic mutations,our strategy em...
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