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新一代基因组编辑技术在基因治疗及生物制药领域中的应用

Application of next generation genome editing technology in gene therapy and biopharmaceuticals

  • 摘要: 近年来,随着锌指核酸酶、转录激活子样效应因子核酸酶和成簇可调控间隔短回文重复RNA引导核酸酶的出现,“基因组编辑”技术得到广泛应用。由于此类技术具有高效和可定制的特点,在基因治疗、细胞模型、糖基化工程、细胞工程等方面许多新策略、新方法不断涌现,产生了十分重要的影响。本文就近年来基因组编辑技术的发展及其在基因治疗和生物制药领域的应用进行综述。

     

    Abstract: In recent years, techeniqus as zinc-finger nucleases(ZFNs), transcription activator-like effector nucleases(TALENs)and clustered regulatory interspaced short palindromic repeats CRISPR/Cas-based RNA-guided DNA endonucleases(CRISPR/Cas)have been applied for genomics editing scale. According to their high efficiency and customizable possibility, such techeniques have a significant influence upon the following research aspects, gene therapy, cell model, protein glycoengineer technology, cell engineering technology, and ect. , which have also been promoting several novel strategies and study methods involved rapidly. This review summerizes the development of genome editing technology and its practical application inside the field of gene therapy and biophamarceutical industry.

     

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