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Review Article

CRISPR与化学工程之间的协同作用

卷 19, 期 3, 2019

页: [147 - 171] 页: 25

弟呕挨: 10.2174/1566523219666190701100556

价格: $65

摘要

由于CRISPR技术的发展，近年来基因治疗和转基因研究迅速发展。 CRISPR技术的快速发展已在很大程度上受益于化学工程。第一，化学或合成物质能够对Cas9或dCas9活性进行时空上和条件控制。它可以防止CRISPR组件的泄漏表达，并最大程度地降低毒性和脱靶效应。多输入逻辑运算和复杂的遗传电路也可以通过靶基因的多重和正交调节来实现。第二，对sgRNA进行合理的化学修饰可通过改善sgRNA的稳定性和对靶基因组位点的结合亲和力来提高基因编辑效率和特异性，从而减少脱靶错配和系统免疫原性。化学修饰的Cas9 mRNA也比天然mRNA具有更高的活性和更低的免疫原性。第三，非病毒载体可通过递送Cas9-sgRNA核糖核蛋白复合物或大型Cas9表达质粒来规避与病毒包装和生产相关的挑战。多功能纳米载体可克服多种生理障碍，实现针对配体的细胞摄取和血脑屏障穿越，从而增强体内基因组编辑。化学工程还可以通过改善载体内在化，允许组织特异性转基因表达并防止体内病毒载体失活来促进基于病毒的传递。这篇综述旨在讨论化学工程如何帮助改善现有的CRISPR应用并为生物医学研究提供新技术。还强调了每种化学工程方法的有用性，优势和分子作用。

关键词: CRISPR

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图形摘要

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