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球形核酸纳米粒子:治疗潜力。

Spherical Nucleic Acid Nanoparticles: Therapeutic Potential.

机构信息

Biomedical Engineering, University of Delaware, Newark, DE, 19716, USA.

Materials Science and Engineering, University of Delaware, Newark, DE, 19716, USA.

出版信息

BioDrugs. 2018 Aug;32(4):297-309. doi: 10.1007/s40259-018-0290-5.

Abstract

Spherical nucleic acids (SNAs) are highly oriented, well organized, polyvalent structures of nucleic acids conjugated to hollow or solid core nanoparticles. Because they can transfect many tissue and cell types without toxicity, induce minimum immune response, and penetrate various biological barriers (such as the skin, blood-brain barrier, and blood-tumor barrier), they have become versatile tools for the delivery of nucleic acids, drugs, and proteins for various therapeutic purposes. This article describes the unique structures and properties of SNAs and discusses how these properties enable their application in gene regulation, immunomodulation, and drug and protein delivery. It also summarizes current efforts towards clinical translation of SNAs and provides an expert opinion on remaining challenges to be addressed in the path forward to the clinic.

摘要

球形核酸(SNAs)是一种高度定向、组织良好、多价的核酸结构,与中空或实心核纳米颗粒相连。由于它们可以转染多种组织和细胞类型而没有毒性,诱导最小的免疫反应,并穿透各种生物屏障(如皮肤、血脑屏障和血肿瘤屏障),因此它们已成为用于各种治疗目的的核酸、药物和蛋白质传递的多功能工具。本文描述了 SNAs 的独特结构和特性,并讨论了这些特性如何使其能够应用于基因调控、免疫调节以及药物和蛋白质的传递。它还总结了目前将 SNAs 推向临床应用的努力,并就未来临床应用中需要解决的挑战提供了专家意见。

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