Lipid Nanoparticle–mRNA Formulations for Therapeutic Applications,Accounts of Chemical Research

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Lipid Nanoparticle–mRNA Formulations for Therapeutic Applications
Accounts of Chemical Research ( IF 16.4 ) Pub Date : 2021-11-18 , DOI: 10.1021/acs.accounts.1c00550
Chang Wang ₁ , Yuebao Zhang ₁ , Yizhou Dong _{1,

2}

Affiliation

After decades of extensive fundamental studies and clinical trials, lipid nanoparticles (LNPs) have demonstrated effective mRNA delivery such as the Moderna and Pfizer-BioNTech vaccines fighting against COVID-19. Moreover, researchers and clinicians have been investigating mRNA therapeutics for a variety of therapeutic indications including protein replacement therapy, genome editing, and cancer immunotherapy. To realize these therapeutics in the clinic, there are many formidable challenges. First, novel delivery systems such as LNPs with high delivery efficiency and low toxicity need to be developed for different cell types. Second, mRNA molecules need to be engineered for improved pharmaceutical properties. Lastly, the LNP–mRNA nanoparticle formulations need to match their therapeutic applications.

中文翻译：

用于治疗应用的脂质纳米颗粒-mRNA 制剂

经过数十年的广泛基础研究和临床试验，脂质纳米颗粒 (LNP) 已证明可有效递送 mRNA，例如 Moderna 和 Pfizer-BioNTech 疫苗可对抗 COVID-19。此外，研究人员和临床医生一直在研究用于各种治疗适应症的 mRNA 疗法，包括蛋白质替代疗法、基因组编辑和癌症免疫疗法。要在临床上实现这些疗法，存在许多艰巨的挑战。首先，需要针对不同的细胞类型开发新型递送系统，例如具有高递送效率和低毒性的 LNP。其次，需要对 mRNA 分子进行改造以改善药物特性。最后，LNP-mRNA 纳米颗粒配方需要与其治疗应用相匹配。

更新日期：2021-12-07

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