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信使核糖核酸纳米药物的生物医学应用。

Biomedical applications of mRNA nanomedicine.

作者信息

Xiong Qingqing, Lee Gha Young, Ding Jianxun, Li Wenliang, Shi Jinjun

机构信息

Center for Nanomedicine and Department of Anesthesiology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.

Department of Hepatobiliary Cancer, Tianjin Medical University Cancer Institute & Hospital, Tianjin 300060, China.

出版信息

Nano Res. 2018 Oct;11(10):5281-5309. doi: 10.1007/s12274-018-2146-1. Epub 2018 Jul 27.

Abstract

As an attractive alternative to plasmid DNA, messenger RNA (mRNA) has recently emerged as a promising class of nucleic acid therapeutics for biomedical applications. Advances in addressing the inherent shortcomings of mRNA and in the development of nanoparticle-based delivery systems have prompted the development and clinical translation of mRNA-based medicines. In this review, we discuss the chemical modification strategies of mRNA to improve its stability, minimize immune responses, and enhance translational efficacy. We also highlight recent progress in nanoparticle-based mRNA delivery. Considerable attention is given to the increasingly widespread applications of mRNA nanomedicine in the biomedical fields of vaccination, protein-replacement therapy, gene editing, and cellular reprogramming and engineering.

摘要

作为质粒DNA颇具吸引力的替代物,信使核糖核酸(mRNA)最近已成为一类用于生物医学应用的、前景广阔的核酸疗法。在解决mRNA固有缺点以及基于纳米颗粒的递送系统研发方面取得的进展,推动了基于mRNA的药物的研发和临床转化。在本综述中,我们讨论了mRNA的化学修饰策略,以提高其稳定性、将免疫反应降至最低并增强翻译效率。我们还强调了基于纳米颗粒的mRNA递送方面的最新进展。mRNA纳米药物在疫苗接种、蛋白质替代疗法、基因编辑以及细胞重编程和工程等生物医学领域日益广泛的应用受到了相当多的关注。

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