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基于小干扰RNA的纳米疗法在癌症治疗中的临床进展

Clinical Advances of siRNA-Based Nanotherapeutics for Cancer Treatment.

作者信息

Hattab Dima, Gazzali Amirah Mohd, Bakhtiar Athirah

机构信息

Faculty of Pharmacy, University of Jordan, Queen Rania Street, Amman 11942, Jordan.

School of Pharmaceutical Sciences, Universiti Sains Malaysia, Penang 11800, Malaysia.

出版信息

Pharmaceutics. 2021 Jul 2;13(7):1009. doi: 10.3390/pharmaceutics13071009.

Abstract

Cancer is associated with single or multiple gene defects. Recently, much research has focused on incorporating genetic materials as one of the means to treat various types of carcinomas. RNA interference (RNAi) conveys an alternative genetic approach for cancer patients, especially when conventional medications fail. RNAi involves the inhibition of expression of specific messenger RNA that signals for uncontrollable cell growth and proliferation, most notably with carcinoma cells. This molecular technology is promising as genetic materials allow us to overcome issues associated with chemotherapeutic agents including organ damage associated with severe drug toxicities. Nonetheless, vast challenges impede successful gene therapy application, including low tumor localization, low stability and rapid clearance from the blood circulation. Owing to the limited treatment opportunities for the management of cancer, the development of effective siRNA carrier systems involving nanotherapeutics has been extensively explored. Over the past years, several siRNA nanotherapeutics have undergone a period of clinical investigation, with some demonstrating promising antitumor activities and safety profiles. Extensive observation of siRNA-nanoparticles is necessary to ensure commercial success. Therefore, this review mainly focuses on the progress of siRNAs-loaded nanoparticles that have undergone clinical trials for cancer treatment. The status of the siRNA nanotherapeutics is discussed, allowing comprehensive understanding of their gene-mediated therapeutics.

摘要

癌症与单个或多个基因缺陷相关。最近,许多研究都集中在将遗传物质作为治疗各种类型癌症的手段之一。RNA干扰(RNAi)为癌症患者提供了一种替代的遗传方法,尤其是在传统药物失效时。RNAi涉及抑制特定信使RNA的表达,该信使RNA指示不可控的细胞生长和增殖,在癌细胞中尤为明显。这种分子技术很有前景,因为遗传物质使我们能够克服与化疗药物相关的问题,包括与严重药物毒性相关的器官损伤。尽管如此,巨大的挑战阻碍了基因治疗的成功应用,包括肿瘤定位低、稳定性差和从血液循环中快速清除。由于癌症治疗机会有限,涉及纳米疗法的有效siRNA载体系统的开发已得到广泛探索。在过去几年中,几种siRNA纳米疗法已经历了临床研究阶段,一些显示出有前景的抗肿瘤活性和安全性。对siRNA纳米颗粒进行广泛观察对于确保商业成功至关重要。因此,本综述主要关注已进行癌症治疗临床试验的负载siRNA的纳米颗粒的进展。讨论了siRNA纳米疗法的现状,以便全面了解其基因介导的治疗方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4a19/8309123/09ceb4af1ada/pharmaceutics-13-01009-g001.jpg

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