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    • CPSF3-dependent Pre-Mrna Processing As a Druggable Node in AML and Ewing's Sarcoma

    Nature Chemical Biology（2020）SCI 1区

    Novartis Institutes for BioMedical Research

    Cited 69|Views57
    Abstract
    An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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    Key words
    Cancer,RNA,Small molecules,Target identification,Transcription,Chemistry/Food Science,general,Biochemical Engineering,Biochemistry,Cell Biology,Bioorganic Chemistry
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    【要点】：本文揭示了CPSF3依赖的剪接预处理在急性髓细胞性白血病（AML）和ewing's肉瘤中作为可药物靶点的作用。
    

    【方法】：研究采用生物信息学和实验生物学相结合的方法，通过分析RNA测序数据，确定了CPSF3在剪接预处理中的作用，并使用CRISPR/Cas9技术进行功能验证。
    

    【实验】：在AML和ewing's肉瘤细胞系中进行了实验，使用了多个细胞系和病人样本的RNA测序数据集，实验结果表明CPSF3的抑制可以显著影响肿瘤细胞的生长和生存。
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