冠状病毒(COVID-19)大流行强调了基于mRNA的疫苗的关键作用,适应性强,易于制造,和安全的预防方法。基于mRNA的治疗正在成为治疗多种疾病的希望途径,包括传染病,癌症,自身免疫性疾病,遗传性疾病,和罕见的疾病。尽管如此,mRNA的体内递送由于其不稳定性而面临挑战,次优交货,以及引发不希望的免疫反应的可能性。在这种情况下,开发有效的药物递送系统,特别是纳米颗粒(NPs),是最重要的。具有生物物理和化学特性,易于表面定制,这些NP已证明在体内的mRNA递送增强,并导致几种基于NP的制剂被批准用于临床.尽管取得了这些进步,发展精致的必要性,有针对性的NP递送系统仍然势在必行。这篇综述全面调查了生物,翻译,NPs介导的mRNA疗法在预防和治疗多种疾病方面的临床进展。通过解决加强现有方法的关键因素,它旨在为精确和有效的基于mRNA的治疗干预措施的未来发展提供信息.
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.