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,Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in h
[期刊论文] 作者：,Wenqin Ying,Yidi Sun,Lu-Lu Lai,Hai-Zhu Chen,Xin-Xin Guo,Qi-Jie Zhang,Shuang Wu,Changyang Zhou,Xiaowen Shen,
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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