Hunan Province Cooperative Innovation Center for Molecular Target New Drug Study, School of Pharmaceutical Science, Hengyang Medical School, University of South China, 421001, Hengyang, P. R. China.
Nanoscale. 2024 Feb 29;16(9):4392-4406. doi: 10.1039/d3nr05971e.
Gene therapy is a revolutionary treatment approach in the 21st century, offering significant potential for disease prevention and treatment. However, the efficacy of gene delivery is often compromised by the inherent challenges of gene properties and vector-related defects. It is crucial to explore ways to enhance the curative effect of gene drugs and achieve safer, more widespread, and more efficient utilization, which represents a significant challenge in amplification gene therapy advancements. Spherical nucleic acids (SNAs), with their unique physicochemical properties, are considered an innovative solution for scalable gene therapy. This review aims to comprehensively explore the amplifying contributions of SNAs in gene therapy and emphasize the contribution of SNAs to the amplification effect of gene therapy from the aspects of structure, application, and recent clinical translation - an aspect that has been rarely reported or explored thus far. We begin by elucidating the fundamental characteristics and scaling-up properties of SNAs that distinguish them from traditional linear nucleic acids, followed by an analysis of combined therapy treatment strategies, theranostics, and clinical translation amplified by SNAs. We conclude by discussing the challenges of SNAs and provide a prospect on the amplification characteristics. This review seeks to update the current understanding of the use of SNAs in gene therapy amplification and promote further research into their clinical translation and amplification of gene therapy.
基因治疗是 21 世纪的一种革命性治疗方法，为疾病的预防和治疗提供了巨大的潜力。然而，基因传递的疗效常常受到基因特性和载体相关缺陷的固有挑战的影响。探索增强基因药物疗效并实现更安全、更广泛、更有效的利用途径，是放大基因治疗进展所面临的重大挑战。球形核酸（SNA）具有独特的理化性质，被认为是可扩展基因治疗的创新解决方案。本综述旨在全面探讨 SNA 在基因治疗中的放大作用，并从结构、应用和最近的临床转化等方面强调 SNA 对基因治疗放大效应的贡献，这是迄今为止很少有报道或探索的方面。我们首先阐明了 SNA 的基本特征和可扩展性质，这些性质使其有别于传统的线性核酸，然后分析了 SNA 放大的联合治疗策略、治疗诊断学和临床转化。最后，我们讨论了 SNA 的挑战，并对其放大特性进行了展望。本综述旨在更新当前对 SNA 在基因治疗放大中的应用的理解，并促进其临床转化和基因治疗放大的进一步研究。
