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靶向治疗 RNA 疗法新时代的儿科实体肿瘤。

Targeting pediatric solid tumors in the new era of RNA therapeutics.

机构信息

Musculoskeletal Science and Translational Research Center, Department of Orthopedics, Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, Thailand; Center of Multidisciplinary Technology for Advanced Medicine (CMUTEAM), Faculty of Medicine, Chiang Mai University, Chiang Mai 50200, Thailand; Extracellular Vesicle Working Group, University of Cincinnati College of Medicine, Cincinnati, OH 45267, USA.

Department of Biomedical Science, Faculty of Medicine, Prince of Songkla University, Songkhla, Thailand; Department of Surgery, Faculty of Medicine, Prince of Songkla University, Songkhla, Thailand; Extracellular Vesicle Working Group, University of Cincinnati College of Medicine, Cincinnati, OH 45267, USA.

出版信息

Crit Rev Oncol Hematol. 2024 Aug;200:104406. doi: 10.1016/j.critrevonc.2024.104406. Epub 2024 Jun 2.

Abstract

Despite substantial progress in pediatric cancer treatment, poor prognosis remained for patients with recurrent or metastatic disease, given the limitations of approved targeted treatments and immunotherapies. RNA therapeutics offer significant potential for addressing a broad spectrum of diseases, including cancer. Advances in manufacturing and delivery systems are paving the way for the rapid development of therapeutic RNAs for clinical applications. This review summarizes therapeutic RNA classifications and the mechanisms of action, highlighting their potential in manipulating major cancer-related pathways and biological effects. We also focus on the pre-clinical investigation of RNA molecules with efficient delivery systems for their therapeutic potential targeting pediatric solid tumors.

摘要

尽管在儿科癌症治疗方面取得了重大进展,但鉴于批准的靶向治疗和免疫疗法的局限性,复发或转移性疾病患者的预后仍然较差。RNA 疗法为治疗包括癌症在内的广泛疾病提供了巨大的潜力。制造和输送系统的进步为临床应用的治疗性 RNA 的快速发展铺平了道路。这篇综述总结了治疗性 RNA 的分类和作用机制,强调了它们在操纵主要癌症相关途径和生物学效应方面的潜力。我们还重点介绍了具有高效输送系统的 RNA 分子的临床前研究,以探讨其针对儿科实体瘤的治疗潜力。

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