as9核糖核蛋白递送的研究进展
Research Progress of Cas9 Ribonucleoprotein Delivery
簇状规则间隔短回文重复序列(CRISPR)/CRISPR相关系统是一种革命性的基因编辑技术,它为科学研究提供了强大的工具,在治疗各种疾病方面具有巨大的潜力。近年来,随着基因编辑技术的快速发展,CRISPR/Cas基因编辑系统成为继锌指核酸酶(ZFNs)系统和转录激活物样效应核酸酶(TALENs)系统之后的新一代靶向基因编辑技术之一。CRISPR 相关蛋白9(CRISPR/Cas9)可适用于使用定制的 CRISPR RNA (crRNA) 以及常见的反式激活 CRISPR RNA (tracrRNA) 或作为嵌合 crRNA-tracrRNA 杂合体的人工单向导 RNA (sgRNA) 进行基因组编辑,但由于递送的CRISPR/Cas9未能高效地递送到靶细胞,使得这种生物技术向治疗的转化仍面临不小的挑战。本文将对目前Cas9核糖核蛋白(RNP)的递送方式,包括物理途径递送、病毒载体以及非病毒载体途径递送进行讨论,并对这些在细胞内递送 Cas9 RNP 的递送方式的发展前景进行了展望,以期为合理设计下一代递送材料提供新思路。
he clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) system as a revolutionary genome editing technology offers a powerful tool for scientific research and has great potential in the treatment of various diseases. As the development in gene editing technology progresses rapidly in recent years, CRISPR/Cas gene editing system emerged as one of the new generation of targeted genome editing technologies, alongside the zinc finger nucleases (ZFNs) system and transcription activator-like effector nucleases (TALENs) system.The CRISPR-associate protein9can be adapted to genome editing with a customized CRISPR RNA (crRNA) together with a common transactivating CRISPR RNA (tracrRNA) or an artificial single guide RNA (sgRNA) which was a chimeric crRNA-tracrRNA hybrid. However, since the delivered CRISPR/Cas9 cannot be efficiently delivered to the target cells, the translation of this biotechnology into therapy still faces considerable challenges. This paper will discuss the current delivery methods of Cas9 ribonucleoprotein (RNP), including physical delivery, viral vector and non-viral vector delivery, and prospect the development prospects of these delivery methods for intracellular delivery of Cas9 RNP, in order to provide a rationale for designing Generation materials provide theoretical reference.
林珊、冯旭利
生物科学研究方法、生物科学研究技术基础医学生物工程学
纳米材料药物输送基因编辑CRISPR核糖核蛋白
Nanomaterialsrug deliveryGene editingRISPRRibonucleoproteins
林珊,冯旭利.as9核糖核蛋白递送的研究进展[EB/OL].(2022-03-24)[2025-05-28].http://www.paper.edu.cn/releasepaper/content/202203-362.点此复制
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