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Rhabdomyolysis and respiratory insufficiency due to the common ETFDH mutation of c.250G＞A in two pat
[期刊论文] 作者：Hai-Zhu Chen,Ming Jin,Nai-Qing,
来源：中华医学杂志（英文版） 年份：2019
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[期刊论文] 作者：Liang-Liang Qiu,Xiao-Dan Lin,Guo-Rong Xu,Li-Li Wang,Zhi-Xian Ye,Feng Lin,Hai-Zhu Chen,Min-Ting Lin,Nai-Qing,
来源：中华医学杂志英文版 年份：2021
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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：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,
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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