Aptamer-Based Targeted Delivery of Functional Nucleic Acids,Journal of the American Chemical Society

当前位置： X-MOL 学术 › J. Am. Chem. Soc. › 论文详情

Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)

Aptamer-Based Targeted Delivery of Functional Nucleic Acids
Journal of the American Chemical Society ( IF 14.4 ) Pub Date : 2023-03-29 , DOI: 10.1021/jacs.3c00841
Sitao Xie _{1,

2} , Weidi Sun _{1,

2} , Ting Fu _{1,

3} , Xiangsheng Liu _{1,

3} , Peng Chen ₁ , Liping Qiu _{1,

2} , Fengli Qu _{1,

4} , Weihong Tan _{1,

2,

5}

Affiliation

Functional nucleic acid (NA)-based drugs have a broad range of applications since they allow the alteration and control of gene/protein expression patterns in cells. In principle, functional NAs need to be transported precisely and efficiently to target cells to guarantee both functionality and safety. Owing to their negative charges, it is difficult for natural NAs to cross the cell membrane composed of lipid bilayer and enter targeted cells. Worse still, the delivery of undirected functional NAs to nontargeted healthy cells and/or tissues would induce unpredictable adverse effects. Therefore, the precisely targeted delivery of functional NAs to specific cells/organs, particularly in extrahepatic sites, is required. Since aptamers can bind to various proteins on the cell surface with high specificity and selectivity, they can serve as the molecular recognition units to accurately bind target cells and subsequently enable the efficient delivery of cargo. In this perspective, we summarize the original, proof-of-concept aptamer-based strategies for the targeted delivery of functional NAs. A few specific examples are then discussed, followed by our perspectives on some of the challenges and opportunities that lie ahead.

中文翻译：

基于适体的功能性核酸靶向递送

基于功能性核酸 (NA) 的药物具有广泛的应用，因为它们允许改变和控制细胞中的基因/蛋白质表达模式。原则上，功能性 NA 需要准确高效地运输到目标细胞，以保证功能性和安全性。由于它们的负电荷，天然 NAs 很难穿过由脂质双层组成的细胞膜并进入目标细胞。更糟糕的是，将未定向的功能性 NA 递送至非靶向健康细胞和/或组织会引起不可预测的副作用。因此，需要将功能性 NA 精确靶向递送至特定细胞/器官，特别是在肝外部位。由于适配体可以高度特异性和选择性地结合细胞表面的各种蛋白质，它们可以作为分子识别单元，准确结合靶细胞，随后实现货物的有效运送。从这个角度来看，我们总结了用于功能性 NA 靶向递送的原始的、基于概念验证的适体策略。然后讨论了一些具体的例子，然后是我们对未来的一些挑战和机遇的看法。

更新日期：2023-03-29

点击分享查看原文

点击收藏

阅读更多本刊新发论文本刊介绍/投稿指南