Nanomedicine-Based Approaches for mRNA Delivery.,Molecular Pharmaceutics

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Nanomedicine-Based Approaches for mRNA Delivery.
Molecular Pharmaceutics ( IF 4.5 ) Pub Date : 2020-08-26 , DOI: 10.1021/acs.molpharmaceut.0c00618
Satoshi Uchida _{1,

2} , Federico Perche ₃ , Chantal Pichon _{3,

4} , Horacio Cabral _{1,

2}

Affiliation

Messenger RNA (mRNA) has immense potential for developing a wide range of therapies, including immunotherapy and protein replacement. As mRNA presents no risk of integration into the host genome and does not require nuclear entry for transfection, which allows protein production even in nondividing cells, mRNA-based approaches can be envisioned as safe and practical therapeutic strategies. Nevertheless, mRNA presents unfavorable characteristics, such as large size, immunogenicity, limited cellular uptake, and sensitivity to enzymatic degradation, which hinder its use as a therapeutic agent. While mRNA stability and immunogenicity have been ameliorated by direct modifications on the mRNA structure, further improvements in mRNA delivery are still needed for promoting its activity in biological settings. In this regard, nanomedicine has shown the ability for spatiotemporally controlling the function of a myriad of bioactive agents in vivo. Direct engineering of nanomedicine structures for loading, protecting, and releasing mRNA and navigating in biological environments can then be applied for promoting mRNA translation toward the development of effective treatments. Here, we review recent approaches aimed at enhancing mRNA function and its delivery through nanomedicines, with particular emphasis on their applications and eventual clinical translation.

中文翻译：

基于纳米医学的 mRNA 递送方法。

信使 RNA (mRNA) 具有开发多种疗法的巨大潜力，包括免疫疗法和蛋白质替代疗法。由于 mRNA 没有整合到宿主基因组中的风险，并且不需要进入细胞核进行转染，即使在非分裂细胞中也可以产生蛋白质，因此可以将基于 mRNA 的方法设想为安全实用的治疗策略。然而，mRNA 呈现出不利的特征，例如大尺寸、免疫原性、有限的细胞摄取和对酶促降解的敏感性，这阻碍了其作为治疗剂的使用。虽然 mRNA 的稳定性和免疫原性已通过对 mRNA 结构的直接修饰得到改善，但仍需要进一步改进 mRNA 递送以促进其在生物环境中的活性。在这方面，体内。用于加载、保护和释放 mRNA 以及在生物环境中导航的纳米药物结构的直接工程可用于促进 mRNA 翻译以开发有效的治疗方法。在这里，我们回顾了最近旨在通过纳米药物增强 mRNA 功能及其传递的方法，特别强调它们的应用和最终的临床转化。

更新日期：2020-10-05

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