Investigation of in vitro delivery conditions for self-amplifying mRNA.

IF 3.6 3区生物学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Bioprocess and Biosystems Engineering Pub Date : 2025-11-01 Epub Date: 2025-08-05 DOI:10.1007/s00449-025-03215-4

Wenting Li, Yiming Wang, Yi-Xin Huo, Yuan Lu

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Abstract

Self-amplifying mRNA (SAM) shows promise for vaccines and gene therapy because of its self-replicating ability. However, current studies lack sufficient information for systematic parameter optimization and differentiation from conventional non-replicating mRNA (NRM). Therefore, the transfection efficiency of NRM and SAM platforms was evaluated by comparing delivery vectors and optimizing parameters for the SAM protocol. SAM and NRM showed similar transfection preferences, but their efficiencies differed. Optimized SAM transfection parameters were then established, including dose and incubation time. In this study, an in vitro multi-parameter delivery system for SAM was constructed, providing valuable insights into SAM transfection and its distinction from regular mRNA. This study contributes an experimental basis for the rational screening of nucleic acid drug carriers and the establishment of SAM multi-parameter evaluation criteria, and also lays an important foundation for optimizing low-dose immunization strategies and their clinical application translation.

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自扩增mRNA体外递送条件的研究。

自扩增mRNA （SAM）因其自我复制能力而在疫苗和基因治疗中显示出前景。然而，目前的研究缺乏足够的信息来优化系统参数并与传统的非复制mRNA （NRM）进行区分。因此，通过比较传递载体和优化SAM协议参数，评估NRM和SAM平台的转染效率。SAM和NRM表现出相似的转染偏好，但它们的效率不同。建立最佳的SAM转染参数，包括转染剂量和孵育时间。本研究构建了SAM体外多参数传递系统，为SAM转染及其与常规mRNA的区别提供了有价值的见解。本研究为合理筛选核酸药物载体和建立SAM多参数评价标准提供了实验依据，也为优化低剂量免疫策略及其临床应用转化奠定了重要基础。

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来源期刊

Bioprocess and Biosystems Engineering 工程技术-工程：化工

CiteScore

7.90

自引率

2.60%

发文量

147

审稿时长

2.6 months

期刊介绍： Bioprocess and Biosystems Engineering provides an international peer-reviewed forum to facilitate the discussion between engineering and biological science to find efficient solutions in the development and improvement of bioprocesses. The aim of the journal is to focus more attention on the multidisciplinary approaches for integrative bioprocess design. Of special interest are the rational manipulation of biosystems through metabolic engineering techniques to provide new biocatalysts as well as the model based design of bioprocesses (up-stream processing, bioreactor operation and downstream processing) that will lead to new and sustainable production processes. Contributions are targeted at new approaches for rational and evolutive design of cellular systems by taking into account the environment and constraints of technical production processes, integration of recombinant technology and process design, as well as new hybrid intersections such as bioinformatics and process systems engineering. Manuscripts concerning the design, simulation, experimental validation, control, and economic as well as ecological evaluation of novel processes using biosystems or parts thereof (e.g., enzymes, microorganisms, mammalian cells, plant cells, or tissue), their related products, or technical devices are also encouraged. The Editors will consider papers for publication based on novelty, their impact on biotechnological production and their contribution to the advancement of bioprocess and biosystems engineering science. Submission of papers dealing with routine aspects of bioprocess engineering (e.g., routine application of established methodologies, and description of established equipment) are discouraged.