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用于下一代疗法的转染技术。

Transfection Technologies for Next-Generation Therapies.

作者信息

Simkhada Dinesh, Teo Su Hui Catherine, Deorkar Nandu, Vemuri Mohan C

机构信息

Biopharma and Bioproduction, Cell and Gene Therapy, Avantor, 77 Corporate Drive, Bridgewater, NJ 08807, USA.

出版信息

J Clin Med. 2025 Aug 5;14(15):5515. doi: 10.3390/jcm14155515.

Abstract

: Transfection is vital for gene therapy, mRNA treatments, CAR-T cell therapy, and regenerative medicine. While viral vectors are effective, non-viral systems like lipid nanoparticles (LNPs) offer safer, more flexible alternatives. This work explores emerging non-viral transfection technologies to improve delivery efficiency and therapeutic outcomes. : This review synthesizes the current literature and recent advancements in non-viral transfection technologies. It focuses on the mechanisms, advantages, and limitations of various delivery systems, including lipid nanoparticles, biodegradable polymers, electroporation, peptide-based carriers, and microfluidic platforms. Comparative analysis was conducted to evaluate their performance in terms of transfection efficiency, cellular uptake, biocompatibility, and potential for clinical translation. Several academic search engines and online resources were utilized for data collection, including Science Direct, PubMed, Google Scholar Scopus, the National Cancer Institute's online portal, and other reputable online databases. : Non-viral systems demonstrated superior performance in delivering mRNA, siRNA, and antisense oligonucleotides, particularly in clinical applications. Biodegradable polymers and peptide-based systems showed promise in enhancing biocompatibility and targeted delivery. Electroporation and microfluidic systems offered precise control over transfection parameters, improving reproducibility and scalability. Collectively, these innovations address key challenges in gene delivery, such as stability, immune response, and cell-type specificity. : The continuous evolution of transfection technologies is pivotal for advancing gene and cell-based therapies. Non-viral delivery systems, particularly LNPs and emerging platforms like microfluidics and biodegradable polymers, offer safer and more adaptable alternatives to viral vectors. These innovations are critical for optimizing therapeutic efficacy and enabling personalized medicine, immunotherapy, and regenerative treatments. Future research should focus on integrating these technologies to develop next-generation transfection platforms with enhanced precision and clinical applicability.

摘要

转染对于基因治疗、mRNA治疗、CAR-T细胞治疗和再生医学至关重要。虽然病毒载体有效,但脂质纳米颗粒(LNP)等非病毒系统提供了更安全、更灵活的替代方案。这项工作探索新兴的非病毒转染技术,以提高递送效率和治疗效果。

本综述综合了非病毒转染技术的当前文献和最新进展。它重点关注各种递送系统的机制、优点和局限性,包括脂质纳米颗粒、可生物降解聚合物、电穿孔、基于肽的载体和微流体平台。进行了比较分析,以评估它们在转染效率、细胞摄取、生物相容性和临床转化潜力方面的性能。利用了几个学术搜索引擎和在线资源进行数据收集,包括Science Direct、PubMed、Google Scholar Scopus、美国国立癌症研究所的在线门户以及其他知名在线数据库。

非病毒系统在递送mRNA、siRNA和反义寡核苷酸方面表现出卓越性能,尤其是在临床应用中。可生物降解聚合物和基于肽的系统在增强生物相容性和靶向递送方面显示出前景。电穿孔和微流体系统对转染参数提供了精确控制,提高了可重复性和可扩展性。总体而言,这些创新解决了基因递送中的关键挑战,如稳定性、免疫反应和细胞类型特异性。

转染技术的持续发展对于推进基于基因和细胞的治疗至关重要。非病毒递送系统,特别是LNP以及微流体和可生物降解聚合物等新兴平台,为病毒载体提供了更安全、更具适应性的替代方案。这些创新对于优化治疗效果以及实现个性化医学、免疫疗法和再生治疗至关重要。未来的研究应专注于整合这些技术,以开发具有更高精度和临床适用性的下一代转染平台。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c443/12347970/5feb35443055/jcm-14-05515-g003.jpg

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