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您的检索式：作者名="LAI Qifang"
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| 1 | 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 |
| 2 | Effects of Seawater Acidification on Early Development of Clam Cyclina sinensis显示文摘Anthropogenic emission of atmospheric carbon dioxide (CO2) has led to a rapid increase in atmospheric CO2 concentra- tion. Increasing atmospheric CO2 can reduce seawater pH and carbonate ions, which may adversely affect the survival of the larvae of calcareous animals. Cyclina sinensis is a commercially and ecologically important species in several Asian countries. Living in coast shallow waters, this species has experienced the coastal environmental changes frequently throughout its life cycle. In this study, we simulated possible future seawater pH values including 8.2, 7.8 and 7.4 and examined the effects of ocean acidification on the early development of C. sinensis. Clam embryos were incubated for 48 h (2 d) in control and high-CO2 seawater to compare embryo- genesis, larval growth and swimming behavior. Fertilization rate was quite sensitive to pH, and moderate acidification could induce a significant decrease in fertilization rate. However, only extreme acidification could bring significant negative effect to hatching rate, body size, and average path velocity of trochophora. Moreover, with seawater acidification, C. sinensis needs much more time to reach the same developmental stage, which increases the risk of larva survival. Together with recent studies demonstrating negative impacts of high CO2 on fertilization and larva swimming behavior, the results imply a future decrease of C. sinensis populations in oceans if its acclimation to the predicted environmental alteration does not occur. | SUI Yanming ZHOU Kai LAI Qifang YAO Zongli GAO Pengchen | 2019 | Journal of Ocean University of China2019，18，4： | 1 |
| 3 | Organocatalytic difluorobenzylation of 1,2-diketones via mild cleavage of carbon-carbon bonds显示文摘α-Aryl-α,α-difluoroacetophenones(DFAPs)are developed as a new class of difluorobenzylation reagents that can be facilely prepared from readily available and cheap starting materials.In situ carbon-carbon bond cleavage of electron-deficient DFAPs gives difluorobenzyl carbanions that undergo base-catalyzed nucleophilic addition to isatins. | Yong Zhang Guo-Wei Lai Long-Jun Nie Qifang He Mei-Juan Lin Rong Chi Dong-Liang Lu Xiaolin Fan | 2022 | Organic Chemistry Frontiers2022，9，3： | 0 |
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