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单粒子电荷检测质谱法能够对脂质纳米颗粒和mRNA包装进行分子表征。

Single particle charge detection mass spectrometry enables molecular characterization of lipid nanoparticles and mRNA packaging.

作者信息

Miller Zachary M, Narsineni Lokesh, Li Yue-Xuan, Gardner Matthew R, Torpey Justin W, Williams Evan R

机构信息

Department of Chemistry, University of California, Berkeley, CA 94720-1460, United States.

Scribe Therapeutics, Alameda, CA 94501, United States.

出版信息

J Control Release. 2025 Aug 10;384:113856. doi: 10.1016/j.jconrel.2025.113856. Epub 2025 May 18.

Abstract

Lipid nanoparticles (LNPs) are effective delivery systems for RNA therapeutics, yet their intrinsic heterogeneity in size and composition make them challenging to characterize. Charge detection mass spectrometry (CDMS) was used to rapidly weigh thousands of individual LNPs. Diameter distributions of empty LNPs from CDMS and cryo-TEM measurements are in excellent agreement demonstrating that these particles are sufficiently stable in the high vacuum environment of the mass spectrometer for accurate mass analysis. A similarly prepared mRNA-packaged LNP sample has a peak mass at ∼70 MDa, 31 MDa higher than that of the empty LNP sample. Four freeze-thaw (FT) cycles of the mRNA-LNPs results in a peak mass at ∼26.5 MDa, indicating significantly degraded LNPs. The degraded LNPs are about 28 % of the population of the mRNA-LNP sample after the first FT cycle. A non-linear least squares fitting routine was developed to convolve the mass distribution of the LNP core with a function that describes the packaging distribution to fit the mRNA-LNP data. Two models of the lipid core mass distribution were used to obtain the distribution of mRNA in the packaged LNPs. These two models provide a lower and upper limit to the average mRNA packaging of 43 and 107 mRNA copies, consistent with a rough estimate of an average of 62 mRNA copies obtained from cryo-TEM images. These results demonstrate the potential for label-free, rapid characterization of mass, diameter, packaging, and stability of LNPs with CDMS.

摘要

脂质纳米颗粒(LNPs)是用于RNA治疗的有效递送系统,但其在大小和组成上固有的异质性使其难以表征。电荷检测质谱(CDMS)被用于快速称量数千个单个的LNPs。来自CDMS和冷冻透射电子显微镜(cryo-TEM)测量的空LNPs的直径分布高度一致,这表明这些颗粒在质谱仪的高真空环境中足够稳定,可进行准确的质量分析。一个经过类似制备的包裹mRNA的LNP样品的峰值质量约为70 MDa,比空LNP样品高31 MDa。mRNA-LNPs的四个冻融(FT)循环导致峰值质量约为26.5 MDa,表明LNPs明显降解。在第一个FT循环后,降解的LNPs约占mRNA-LNP样品总数的28%。开发了一种非线性最小二乘拟合程序,将LNP核心的质量分布与描述包装分布的函数进行卷积,以拟合mRNA-LNP数据。使用脂质核心质量分布的两种模型来获得包装的LNPs中mRNA的分布。这两种模型给出了平均mRNA包装量的下限和上限,分别为43和107个mRNA拷贝,这与从冷冻透射电子显微镜图像获得的平均62个mRNA拷贝的粗略估计一致。这些结果证明了利用CDMS对LNPs的质量、直径、包装和稳定性进行无标记、快速表征的潜力。

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