相关实验视频

Updated: Jun 3, 2025

Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells

Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells

Published on: June 10, 2022

对于mRNA输送的histidylated脂质体的结构和功能表征mRNA输送的histidylated脂质体

Albert Ngalle Loth¹, Manon Maroquenne², Ayoub Medjmedj¹

¹Centre de Biophysique Moléculaire, CBM, CNRS UPR4301, Orléans, France.

Journal of controlled release : official journal of the Controlled Release Society

|January 9, 2025

概括

优化化脂质体 (LYX) 提供稳定,长期的mRNA输送与高封装. 这些LYX脂质体在细胞和动物模型中显示出治疗mRNA应用的前景.

科学领域:

生物技术是生物技术.
纳米医学是一种纳米医学.
药物输送系统药物输送系统

背景情况:

基于脂质的系统,特别是脂质体,对于mRNA疗法至关重要.
基化脂质体以前显示出高效的核酸输送.
优化脂质体准备可以提高治疗应用的稳定性和有效性.

研究的目的:

为了优化化脂质体 (LYX) 的制备,以提高存储稳定性和大小均性.
为了全面描述用于mRNA输送的LYX脂质体.
评估用于治疗性蛋白质生产的LYX-mRNA脂质组的体外和体内性能.

主要方法:

通过冷干燥和挤出优化了脂质体准备.
描述包括尺寸,多分散性,封装效率和RNase保护试验.
在细胞系和初级细胞中的体外转染,以及在小鼠模型中的体内研究.

主要成果:

优化的LYX脂质体表现出优异的储存稳定性 (4°C下1年),具有一致的大小 (150±10nm) 和高封装效率 (~95%).
LYX脂质体保护mRNA免受降解,并表现出转染能力,尽管由于吸收速度较慢和内分体逃逸,其疗效低于商业标准.
编码BMP2和BMP9的mRNA的成功输送导致了功能性蛋白质的产生,并诱导了体外和体外的BMP信号传递.

结论:

优化LYX脂质体代表了稳定有效的mRNA输送平台.
这些脂质体显示出治疗应用的潜力,包括蛋白质替代疗法,具有有前途的体内结果.
进一步开发LYX脂质体可以推进基于mRNA的治疗策略.

关键词:

运输运输运输运输运输运输细胞内体逃脱是什么?基化脂质体的基化脂质体细胞内贩运细胞内贩运脂质复合体的形态和形态.mRNA疗法是一种mRNA疗法.

更多相关视频

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Published on: January 20, 2023

相关概念视频

Regulated mRNA Transport

Regulated mRNA Transport

In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing...

mRNA Stability and Gene Expression

mRNA Stability and Gene Expression

The structure and stability of mRNA molecules regulates gene expression, as mRNAs are a key step in the pathway from gene to protein. In eukaryotes, the half-life of mRNA varies from a few minutes up to several days. mRNA stability is essential in growth and development. The absence of the proteins regulating its stability, such as tristetraprolin in mice, can cause systemic issues, including bone marrow overgrowth, inflammation, and autoimmunity.
Cis-acting Elements involved in mRNA stability

RNA Stability

RNA Stability

Intact DNA strands can be found in fossils, while scientists sometimes struggle to keep RNA intact under laboratory conditions. The structural variations between RNA and DNA underlie the differences in their stability and longevity. Because DNA is double-stranded, it is inherently more stable. The single-stranded structure of RNA is less stable but also more flexible and can form weak internal bonds. Additionally, most RNAs in the cell are relatively short, while DNA can be up to 250 million...