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| 1 | Base editing-mediated splicing correction therapy for spinal muscular atrophy显示文摘Dear Editor,Spinal muscular atrophy(SMA)is a devastating autosomal recessive motor neuron disease.1,2 Infants with more severe forms of type I SMA die before the age of 2 if no intervention is provided.1,2 Spinraza and zolgensma have been approved by the FDA as SMA therapeutics for pediatric patients.1,2 However,as an antisense oligonucleotide(ASO)based therapy,spinraza requires four loading doses,followed by three annual maintenance doses.The patients would be subjected to repeated intrathecal injections in the procedure.Zolgensma is a single-dose gene-replacement therapy for SMA,but unfortunately is unreliable in maintaining a high,stable level of gene expression.These issues have limited the therapeutic effects of the two approved drugs for SMA. | Xiang Lin Haizhu Chen Ying-Qian Lu Shunyan Hong Xinde Hu Yanxia Gao Lu-Lu Lai Jin-Jing Li Zishuai Wang Wenqin Ying Lixiang Ma Ning Wang Erwei Zuo Hui Yang Wan-Jin Chen | 2020 | Cell Research2020，30，6： | 5 |
| 2 | Fabrication and properties of axisynunetric WC/Co functionally graded hard metal via microwave sintering显示文摘 | Xinde Bai Yunhari Ling | 2003 | Material Science Forum2003，，： | 1 |
| 3 | Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice显示文摘We here report a genome-editing strategy to correct spinal muscular atrophy(SMA).Rather than directly targeting the pathogenic exonic mutations,our strategy employed Cas9 and guide-sg RNA for the targeted disruption of intronic splicing-regulatory elements.We disrupted intronic splicing silencers(ISSs,including ISS-N1 and ISS+100)of survival motor neuron(SMN)2,a key modifier gene of SMA,to enhance exon 7 inclusion and full-length SMN expression in SMA iPSCs.Survival of splicing-corrected iPSC-derived motor neurons was rescued with SMN restoration.Furthermore,co-injection of Cas9 mRNA from Streptococcus pyogenes(SpCas9)or Cas9 from Staphylococcus aureus(SaCas9)alongside their corresponding sgRNAs targeting ISS-N1 into zygotes rescued 56%and 100%of severe SMA transgenic mice(Smn-/-,SMN2tg/-).The median survival of the resulting mice was extended to>400 days.Collectively,our study provides proof-of-principle for a new strategy to therapeutically intervene in SMA and other RNA-splicing-related diseases. | 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 | 2020 | National Science Review2020，7，1： | 1 |
| 4 | Frequency and space representation in the primary auditory cortex of the frequency modulating batEptesicus fuscus显示文摘 | Philip H. -S. Jen Xinde Sun Paul J. J. Lin | 1989 | Journal of Comparative Physiology A1989，，1： | 1 |
| 5 | Fabrication and properties of axisymmetric WC/Co functionally graded hard metal via microwave sintering显示文摘 | Lin Wei Bai Xinde Ling Yunhan | 2003 | Materials Science Forum2003，，： | 1 |
| 6 | The study of combustion synthesis of fine-particle γ-lithium aluminate显示文摘 | Wei Lin Xinde Bai Yunhan Ling Jinlong Yang Wenjun Ma | 2003 | Journal of Materials Science2003，，18： | 1 |
| 7 | Fabrication and properties of axisymmetric WC/Co functionally graded hard metal via microwave sintering 显示文摘 | Lin Wei Bai Xinde Ling Yunhan | 2003 | Mater Sci Forum2003，，： | 1 |
| 8 | Comparison of Time Interval Statistic and Pulse Shape Discrimination in Fast Neutron Detection Method with Liquid Scintillation Detector Loaded Gd显示文摘Neutron usually appears with gamma,which is requiring the detector has the capability of n gamma discrimination.Pulse shape discrimination(PSD)is a common approach of n,gamma judgment,but required a complex process to select a suitable discrimination factor,and poor performance in the low energy range.A method based on the time interval of adjacent Signals was disrupted and adopted to compare to pulse shape discrimination in the fast neutron detection method.A good agreement between the adopted method and PSD method was achieved,including the total count ratio and neutron count ratio.The comparison proves the correctness of the theoretical derivation and validates the method in practice.The advantages and limitations of the method based on time Interval of adjacent signals were analyzed.The method provides an effective way to confirm the calibration of the neutron detection of a liquid scintillation detector.Also,as a simple way only requiring the time information about events,the described method has large application potential in some case of fast neutron flux or intensity measurement. | Mohammed Adel Al-jumaili Qiang Du Jingjun Zhu Xinde Lin Wenbin Lin Changjian Tang Mustafa Raad Kadhim Haoyang Xing Shukui Liu | 2017 | 应用物理前沿(中英文版)2017，5，1： | 0 |
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