Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing.
Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing.
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用于 CRISPR/Cas9 基因组编辑的 Cas9 核糖核蛋白的递送策略。
DOI：
10.7150/thno.47007
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发表时间：
2021
期刊：
影响因子：
12.4
通讯作者：
Cheng Y
中科院分区：
文献类型：
--
作者：
Zhang S;Shen J;Li D;Cheng Y
近年来，CRISPR/Cas9基因组编辑得到了越来越多的关注，然而，将这种生物技术转化为治疗受到CRISPR/Cas9材料有效递送到靶细胞中的阻碍。作为由Cas9蛋白和单向导RNA（sgRNA）组成的核糖核蛋白（RNP）复合物的CRISPR/Cas9系统的直接递送由于其瞬时基因组编辑和减少脱靶效应的优点而成为用于基因组编辑的强大且广泛的方法。本文综述了目前Cas9 RNP的物理途径和合成载体。综述了这些策略在细胞内Cas9 RNP递送中的机制和有益作用。在刺激响应和有针对性的载体RNP交付的发展的例子突出显示。最后，将讨论当前Cas9 RNP递送系统的挑战以及为这一有前途的领域合理设计下一代材料的前景。
CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/Cas9 system as a ribonucleoprotein (RNP) complex consisting of Cas9 protein and single guide RNA (sgRNA) has emerged as a powerful and widespread method for genome editing due to its advantages of transient genome editing and reduced off-target effects. In this review, we summarized the current Cas9 RNP delivery systems including physical approaches and synthetic carriers. The mechanisms and beneficial roles of these strategies in intracellular Cas9 RNP delivery were reviewed. Examples in the development of stimuli-responsive and targeted carriers for RNP delivery are highlighted. Finally, the challenges of current Cas9 RNP delivery systems and perspectives in rational design of next generation materials for this promising field will be discussed.