Liu Xiao, Zhang Xu, Li Jiulong, Meng Huan
Department of Gastroenterology, Beijing Hospital, National Center of Gerontology, Institute of Geriatrics Medicine, Chinese Academy of Medical Sciences, Beijing, China.
CAS Key Laboratory for Biomedical Effects of Nanomaterials and Nanosafety and CAS Center for Excellence in Nanoscience National Center for Nanoscience and Technology, Beijing, China.
Med Rev (2021). 2023 Sep 20;3(4):356-361. doi: 10.1515/mr-2023-0010. eCollection 2023 Aug.
Lipid-based nanoparticles (LNP) have shown significant progress in delivering mRNA for therapeutics, particularly with the success of coronavirus disease 2019 (COVID-19) vaccines. However, there are still challenges, such as organ-specific targeting, sustained protein expression, immunogenicity, and storage that need to be addressed. Therefore, there is interest in developing additional nano drug delivery systems (DDS) to complement LNP technology. Some of these include polymer, lipid-polymer hybrid, organic/inorganic hybrid nanostructure, and inorganic nanoparticle. In our opinion, LNP technology may not be suitable for every disease scenario in categories such as infection disease, cancer, pulmonary disease, autoimmune disorders and genetic rare disease (among others). This is because different diseases may require distinct administration routes, doses, and treatment durations, as well as considerations for biological barriers that may lower the efficacy and/or exert safety concern. In this perspective, we will highlight the need and potential for enhancing the diversity of nano delivery platforms for mRNA-based nanotherapeutics.
基于脂质的纳米颗粒(LNP)在递送用于治疗的mRNA方面已取得显著进展,尤其是随着2019冠状病毒病(COVID-19)疫苗的成功。然而,仍存在一些挑战,如器官特异性靶向、持续的蛋白质表达、免疫原性和储存等问题需要解决。因此,人们有兴趣开发额外的纳米药物递送系统(DDS)来补充LNP技术。其中一些包括聚合物、脂质-聚合物杂化体、有机/无机杂化纳米结构和无机纳米颗粒。我们认为,LNP技术可能并不适用于传染病、癌症、肺病、自身免疫性疾病和遗传性罕见病(以及其他疾病)等各类疾病的每种情况。这是因为不同的疾病可能需要不同的给药途径、剂量和治疗持续时间,以及对可能降低疗效和/或引发安全问题的生物屏障的考虑。从这个角度来看,我们将强调增强基于mRNA的纳米治疗的纳米递送平台多样性的必要性和潜力。