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[核酸跨越生物屏障的递送]

[Nucleic Acid Delivery across Biological Barriers].

作者信息

Miyata Kanjiro

机构信息

Department of Materials Engineering, Graduate School of Engineering, The University of Tokyo.

出版信息

Yakugaku Zasshi. 2021;141(5):635-639. doi: 10.1248/yakushi.20-00219-2.

Abstract

One of the current critical issues in nucleic acid delivery is the efficient mRNA delivery into target cells, directed toward clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) genome editing. To this end, we have developed a variety of cationic polyaspartamide derivatives with varying side chain structures because they can form nanocomplexes, termed polyplexes, with mRNA through electrostatic interactions. Interestingly, the delivery functions were highly affected by the chemical structures of the polyaspartamide side chains. Therefore, we review our previous research and provide a rationale for designing polypeptides for mRNA delivery.

摘要

核酸递送当前的关键问题之一是将信使核糖核酸(mRNA)高效递送至靶细胞,用于成簇规律间隔短回文重复序列(CRISPR)和CRISPR相关蛋白(Cas)介导的基因组编辑。为此,我们开发了多种具有不同侧链结构的阳离子聚天冬酰胺衍生物,因为它们可以通过静电相互作用与mRNA形成纳米复合物,即多聚体复合物。有趣的是,递送功能受到聚天冬酰胺侧链化学结构的高度影响。因此,我们回顾了之前的研究,并为设计用于mRNA递送的多肽提供了理论依据。

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