用于 SiRNA 递送的基于核酸的集成大分子复合物:最新进展。

IF 1.1 4区 生物学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2024-05-01 DOI:10.1080/15257770.2024.2347499
Dilpreet Singh, Lovedeep Singh, Simranjeet Kaur, Akshita Arora
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引用次数: 0

摘要

小干扰 RNA(siRNA)具有巨大的治疗潜力,它提供了一种精确抑制致病基因的途径。然而,由于核酸酶降解、细胞吸收率低和免疫原性等原因,如何在体内将 siRNA 运送到靶细胞仍是一项艰巨的挑战。本综述探讨了高效递送 siRNA 的核酸基整合大分子复合物的设计和应用方面的最新进展。我们剖析了近年来开发的创新型递送载体,包括基于脂质的纳米颗粒、聚合物载体、树枝状聚合物复合物以及包含刺激响应元件的混合系统,以实现定向和可控释放。本研究评估了生物共轭技术、主动靶向策略和纳米技术驱动的递送平台等方面的进展,以了解它们对增强 siRNA 递送的贡献。报告还探讨了递送系统设计与生物障碍之间复杂的相互作用,强调了 siRNA 疗法从实验室到临床转化过程中的动态进展和仍然存在的障碍。通过对当前策略和新兴技术的全面概述,我们强调了 siRNA 递送系统在个性化医疗中的未来方向和潜在影响。
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Nucleic acids based integrated macromolecular complexes for SiRNA delivery: Recent advancements.

The therapeutic potential of small interfering RNA (siRNA) is monumental, offering a pathway to silence disease-causing genes with precision. However, the delivery of siRNA to target cells in-vivo remains a formidable challenge, owing to degradation by nucleases, poor cellular uptake and immunogenicity. This overview examines recent advancements in the design and application of nucleic acid-based integrated macromolecular complexes for the efficient delivery of siRNA. We dissect the innovative delivery vectors developed in recent years, including lipid-based nanoparticles, polymeric carriers, dendrimer complexes and hybrid systems that incorporate stimuli-responsive elements for targeted and controlled release. Advancements in bioconjugation techniques, active targeting strategies and nanotechnology-enabled delivery platforms are evaluated for their contribution to enhancing siRNA delivery. It also addresses the complex interplay between delivery system design and biological barriers, highlighting the dynamic progress and remaining hurdles in translating siRNA therapies from bench to bedside. By offering a comprehensive overview of current strategies and emerging technologies, we underscore the future directions and potential impact of siRNA delivery systems in personalized medicine.

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来源期刊
Nucleosides, Nucleotides & Nucleic Acids
Nucleosides, Nucleotides & Nucleic Acids 生物-生化与分子生物学
CiteScore
2.60
自引率
7.70%
发文量
91
审稿时长
6 months
期刊介绍: Nucleosides, Nucleotides & Nucleic Acids publishes research articles, short notices, and concise, critical reviews of related topics that focus on the chemistry and biology of nucleosides, nucleotides, and nucleic acids. Complete with experimental details, this all-inclusive journal emphasizes the synthesis, biological activities, new and improved synthetic methods, and significant observations related to new compounds.
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