研究动态
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推进 mRNA 治疗:纳米颗粒输送系统的作用和未来。

Advancing mRNA Therapeutics: The Role and Future of Nanoparticle Delivery Systems.

发表日期:2024 Jul 02
作者: Jiaxuan Li, Yuning Zhang, Yong-Guang Yang, Tianmeng Sun
来源: MOLECULAR PHARMACEUTICS

摘要:

冠状病毒 (COVID-19) 大流行凸显了基于 mRNA 的疫苗作为强大、适应性强、易于制造且安全的预防方法的关键作用。基于 mRNA 的治疗正在成为治疗多种疾病的一种充满希望的途径,包括传染病、癌症、自身免疫性疾病、遗传性疾病和罕见疾病。尽管如此,mRNA 的体内递送仍面临挑战,因为其不稳定、递送欠佳以及可能引发不良免疫反应。在这种背景下,开发有效的药物输送系统,特别是纳米颗粒(NP)至关重要。这些纳米颗粒具有生物物理和化学特性,并且易于表面定制,已证明可增强体内 mRNA 传递,并导致多种基于纳米颗粒的制剂获得临床使用的批准。尽管取得了这些进步,但开发一种精细的、有针对性的纳米颗粒输送系统仍然是当务之急。这篇综述全面调查了 NPs 介导的 mRNA 疗法在预防和治疗多种疾病方面的生物学、转化和临床进展。通过解决增强现有方法的关键因素,它旨在为未来开发精确有效的基于 mRNA 的治疗干预措施提供信息。
The coronavirus (COVID-19) pandemic has underscored the critical role of mRNA-based vaccines as powerful, adaptable, readily manufacturable, and safe methodologies for prophylaxis. mRNA-based treatments are emerging as a hopeful avenue for a plethora of conditions, encompassing infectious diseases, cancer, autoimmune diseases, genetic diseases, and rare disorders. Nonetheless, the in vivo delivery of mRNA faces challenges due to its instability, suboptimal delivery, and potential for triggering undesired immune reactions. In this context, the development of effective drug delivery systems, particularly nanoparticles (NPs), is paramount. Tailored with biophysical and chemical properties and susceptible to surface customization, these NPs have demonstrated enhanced mRNA delivery in vivo and led to the approval of several NPs-based formulations for clinical use. Despite these advancements, the necessity for developing a refined, targeted NP delivery system remains imperative. This review comprehensively surveys the biological, translational, and clinical progress in NPs-mediated mRNA therapeutics for both the prevention and treatment of diverse diseases. By addressing critical factors for enhancing existing methodologies, it aims to inform the future development of precise and efficacious mRNA-based therapeutic interventions.