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核酸纳米结构与细胞的体内相互作用

In Vivo Interactions of Nucleic Acid Nanostructures With Cells.

作者信息

Xiao Yu, Liang Zhihui, Shyngys Moldir, Baekova Aiana, Cheung Suen, Muljadi Mathias Billy, Bai Qianqian, Zeng Lula, Choi Chung Hang Jonathan

机构信息

Department of Biomedical Engineering, The Chinese University of Hong Kong, Shatin, New Territories, Hong Kong.

Center for Neuromusculoskeletal Restorative Medicine, Hong Kong Science Park, Shatin, New Territories, Hong Kong.

出版信息

Adv Mater. 2025 Jan;37(2):e2314232. doi: 10.1002/adma.202314232. Epub 2024 Sep 12.

DOI:10.1002/adma.202314232
PMID:39263835
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11733725/
Abstract

Nucleic acid nanostructures, derived from the assembly of nucleic acid building blocks (e.g., plasmids and oligonucleotides), are important intracellular carriers of therapeutic cargoes widely utilized in preclinical nanomedicine applications, yet their clinical translation remains scarce. In the era of "translational nucleic acid nanotechnology", a deeper mechanistic understanding of the interactions of nucleic acid nanostructures with cells in vivo will guide the development of more efficacious nanomedicines. This review showcases the recent progress in dissecting the in vivo interactions of four key types of nucleic acid nanostructures (i.e., tile-based, origami, spherical nucleic acid, and nucleic acid nanogel) with cells in rodents over the past five years. Emphasis lies on the cellular-level distribution of nucleic acid nanostructures in various organs and tissues and the cellular responses induced by their cellular entry. Next, in the spirit of preclinical translation, this review features the latest interactions of nucleic acid nanostructures with cells in large animals and humans. Finally, the review offers directions for studying the interactions of nucleic acid nanostructures with cells from both materials and biology perspectives and concludes with some regulatory updates.

摘要

核酸纳米结构由核酸构建模块（如质粒和寡核苷酸）组装而成，是治疗性货物重要的细胞内载体，在临床前纳米医学应用中广泛使用，但其临床转化仍然很少。在“转化性核酸纳米技术”时代，对核酸纳米结构与体内细胞相互作用的更深入机制理解将指导更有效的纳米药物的开发。本综述展示了过去五年在剖析四种关键类型的核酸纳米结构（即基于瓦片的、折纸的、球形核酸和核酸纳米凝胶）与啮齿动物细胞的体内相互作用方面的最新进展。重点在于核酸纳米结构在各种器官和组织中的细胞水平分布以及它们进入细胞所诱导的细胞反应。接下来，本着临床前转化的精神，本综述介绍了核酸纳米结构与大型动物和人类细胞的最新相互作用。最后，本综述从材料和生物学角度为研究核酸纳米结构与细胞的相互作用提供了方向，并以一些监管更新作为结论。

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