Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment.

IF 2.7 4区 材料科学 Q3 MATERIALS SCIENCE, MULTIDISCIPLINARY Beilstein Journal of Nanotechnology Pub Date : 2025-03-27 eCollection Date: 2025-01-01 DOI:10.3762/bjnano.16.34
Ana Cubillo Alvarez, Dylan Maguire, Ruairí P Brannigan
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Abstract

This review explores the recent advancements in polymer-assisted delivery systems for antisense oligonucleotides (ASOs) and their potential in precision disease treatment. Synthetic polymers have shown significant promise in enhancing the delivery, stability, and therapeutic efficacy of ASOs by addressing key challenges such as cellular uptake, endosomal escape, and reducing cytotoxicity. The review highlights key studies from the past decade demonstrating how these polymers improve gene silencing efficiencies, particularly in cancer and neurodegenerative disease models. Despite the progress achieved, barriers such as immunogenicity, delivery limitations, and scalability still need to be overcome for broader clinical application. Emerging strategies, including stimuli-responsive polymers and advanced nanoparticle systems, offer potential solutions to these challenges. The review underscores the transformative potential of polymer-enhanced ASO delivery in personalised medicine, emphasising the importance of continued innovation to optimise ASO-based therapeutics for more precise and effective disease treatments.

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合成聚合物辅助反义寡核苷酸递送:精准疾病治疗的靶向方法。
这篇综述探讨了反义寡核苷酸(ASO)聚合物辅助递送系统的最新进展及其在精准疾病治疗中的潜力。合成聚合物通过解决细胞摄取、内体逸出和降低细胞毒性等关键难题,在提高反义寡核苷酸的递送、稳定性和疗效方面显示出巨大的前景。这篇综述重点介绍了过去十年中的主要研究,这些研究证明了这些聚合物是如何提高基因沉默效率的,尤其是在癌症和神经退行性疾病模型中。尽管取得了进展,但要想更广泛地应用于临床,仍需克服免疫原性、递送限制和可扩展性等障碍。新出现的策略,包括刺激响应聚合物和先进的纳米颗粒系统,为应对这些挑战提供了潜在的解决方案。综述强调了聚合物增强型 ASO 给药在个性化医疗中的变革潜力,并强调了持续创新的重要性,以优化基于 ASO 的疗法,从而实现更精确、更有效的疾病治疗。
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来源期刊
Beilstein Journal of Nanotechnology
Beilstein Journal of Nanotechnology NANOSCIENCE & NANOTECHNOLOGY-MATERIALS SCIENCE, MULTIDISCIPLINARY
CiteScore
5.70
自引率
3.20%
发文量
109
审稿时长
2 months
期刊介绍: The Beilstein Journal of Nanotechnology is an international, peer-reviewed, Open Access journal. It provides a unique platform for rapid publication without any charges (free for author and reader) – Platinum Open Access. The content is freely accessible 365 days a year to any user worldwide. Articles are available online immediately upon publication and are publicly archived in all major repositories. In addition, it provides a platform for publishing thematic issues (theme-based collections of articles) on topical issues in nanoscience and nanotechnology. The journal is published and completely funded by the Beilstein-Institut, a non-profit foundation located in Frankfurt am Main, Germany. The editor-in-chief is Professor Thomas Schimmel – Karlsruhe Institute of Technology. He is supported by more than 20 associate editors who are responsible for a particular subject area within the scope of the journal.
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