环状聚(2-甲基-2-恶唑啉)-脂缀合物是用于脂质纳米颗粒mRNA配方的聚(乙二醇)-脂的良好替代品。

IF 5.5 2区 化学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Biomacromolecules Pub Date : 2025-03-10 Epub Date: 2025-02-19 DOI:10.1021/acs.biomac.4c01587
Bianka Golba, Zifu Zhong, Matteo Romio, Ruben Almey, Dieter Deforce, Maarten Dhaenens, Niek N Sanders, Edmondo M Benetti, Bruno G De Geest
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引用次数: 0

摘要

具有可电离阳离子脂质的脂质纳米颗粒(LNPs)彻底改变了RNA药物递送,在基于mrna的治疗方法(如COVID-19疫苗)的成功中发挥了关键作用。这些LNPs的一个重要组成部分是聚乙二醇(PEG)-脂质偶联物,它增强了胶体稳定性,但可能引发抗PEG抗体的产生,导致血液清除加速(ABC)和治疗效果降低。在这项研究中,我们探索了聚(2-甲基-2-恶唑啉)(PMOXA)作为mRNA LNPs的替代稳定剂。我们合成了环状和线性的PMOXA,并将它们与二烷基脂类结合,创造了脂质-聚合物两亲体。我们系统地评估了聚合物拓扑结构如何影响LNPs的物理化学性质,包括体外细胞摄取、转染效率和蛋白冠形成,并直接将这些性质与peg稳定的LNPs进行了比较。小鼠体内实验进一步评估了静脉给药后这些LNPs的生物分布和蛋白质翻译效率。我们的研究结果表明,环状PMOXA偶联物不仅匹配而且可能超过基于peg的类似物的性能,突出了它们作为mRNA-LNP配方的优越替代品的前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Cyclic Poly(2-methyl-2-oxazoline)-Lipid Conjugates Are Good Alternatives to Poly(ethylene glycol)-Lipids for Lipid Nanoparticle mRNA Formulation.

Lipid nanoparticles (LNPs) with ionizable cationic lipids have revolutionized RNA drug delivery, playing a key role in the success of mRNA-based therapeutics, such as COVID-19 vaccines. A vital component of these LNPs is the poly(ethylene glycol) (PEG)-lipid conjugate, which enhances colloidal stability but may trigger the production of anti-PEG antibodies, resulting in accelerated blood clearance (ABC) and diminished therapeutic efficacy. In this study, we explored poly(2-methyl-2-oxazoline) (PMOXA) as an alternative stabilizing agent for mRNA LNPs. We synthesized both cyclic and linear PMOXA, conjugated them to dialkyl lipids, and created lipid-polymer amphiphiles. We systematically evaluated how polymer topology influenced the physicochemical properties of LNPs, including in vitro cellular uptake, transfection efficiency, and protein corona formation, and directly compared these properties with those of PEG-stabilized counterparts. In vivo experiments in mice further assessed the biodistribution and protein translation efficiency of these LNPs following intravenous administration. Our results showed that cyclic PMOXA conjugates not only matched but potentially surpassed the performance of PEG-based analogues, highlighting their promise as a superior alternative in mRNA-LNP formulations.

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来源期刊
Biomacromolecules
Biomacromolecules 化学-高分子科学
CiteScore
10.60
自引率
4.80%
发文量
417
审稿时长
1.6 months
期刊介绍: Biomacromolecules is a leading forum for the dissemination of cutting-edge research at the interface of polymer science and biology. Submissions to Biomacromolecules should contain strong elements of innovation in terms of macromolecular design, synthesis and characterization, or in the application of polymer materials to biology and medicine. Topics covered by Biomacromolecules include, but are not exclusively limited to: sustainable polymers, polymers based on natural and renewable resources, degradable polymers, polymer conjugates, polymeric drugs, polymers in biocatalysis, biomacromolecular assembly, biomimetic polymers, polymer-biomineral hybrids, biomimetic-polymer processing, polymer recycling, bioactive polymer surfaces, original polymer design for biomedical applications such as immunotherapy, drug delivery, gene delivery, antimicrobial applications, diagnostic imaging and biosensing, polymers in tissue engineering and regenerative medicine, polymeric scaffolds and hydrogels for cell culture and delivery.
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