Spherical Nucleic Acid Nanoparticles: Therapeutic Potential.

Spherical Nucleic Acid Nanoparticles: Therapeutic Potential.
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DOI:
10.1007/s40259-018-0290-5
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发表时间:
2018-08
期刊:
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
影响因子:
--
通讯作者:
Day ES
Day ES
中科院分区:
其他
文献类型:
--
作者:
Kapadia CH;Melamed JR;Day ES

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球形核酸 (SNA) 是与空心或实心核心纳米粒子缀合的高度定向、组织良好的多价核酸结构。由于它们可以无毒地转染许多组织和细胞类型,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递送核酸、药物和蛋白质以用于各种治疗目的的多功能工具。本文描述了 SNA 的独特结构和特性,并讨论了这些特性如何使其在基因调控、免疫调节以及药物和蛋白质递送中的应用。它还总结了当前 SNA 临床转化的努力,并就临床道路上仍需解决的剩余挑战提供了专家意见。
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.
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