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mRNA 脂质纳米颗粒在 CAR-T 治疗中的应用:提高疗效的新策略

英文原题:mRNA lipid nanoparticles in CAR-T therapy: a novel strategy to improve efficacy.

查看英文原题

mRNA lipid nanoparticles in CAR-T therapy: a novel strategy to improve efficacy.

PubMed 2025/05/16(内容时间) Nanotechnology Q3 · IF 2.9(JCR 2025)

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中文摘要

CAR-T 细胞免疫疗法治疗血液系统恶性肿瘤已取得显著进展,但仍面临生产流程复杂、成本高和安全性问题等挑战。脂质纳米颗粒(LNP)技术是一种先进的基因递送平台,可凭借高效、低免疫原性和安全性为CAR-T 治疗带来重要进展。LNP使CAR-T 细胞能够在体内生成,从而提高递送效率、降低免疫原性和插入突变风险、简化生产流程并降低成本。LNP的可规模化能力及快速优化特性,使其成为有前景的CAR-T 细胞生产候选平台。LNP技术有望进一步推动CAR-T 免疫疗法发展,并提供更安全、经济的治疗选择。

因此,本文旨在全面系统地综述LNP在CAR-T 治疗中的应用。首先概述CAR-T 疗法的基本设计、流程和当前挑战;随后介绍LNP的特性、其作为基因递送载体的优势,以及其如何提高CAR-T 疗法疗效;最后总结LNP用于CAR-T 治疗的当前研究现状,包括增强T细胞体外转染、在体内原位编程T细胞、促进T细胞活化、减轻CAR-T 疗法副作用,以及将CAR-T 疗法与其他免疫疗法联合。这些进展将有助于设计基于LNP的mRNA递送系统,推动CAR-T 治疗的发展。

展开英文摘要原文

Chimeric antigen receptor T cells (CAR-T) immunotherapy has achieved remarkable progress in the treatment of hematological malignancies.

However, it encounters challenges including complex manufacturing processes, high cost, and safety issues. Lipid nanoparticle (LNP) technology, as an advanced gene delivery platform, offers significant advancements to CAR-T therapy through its high efficiency, low immunogenicity, and safety.

LNP enable in vivo production of CAR-T cells, thereby improving delivery efficiency, reducing the risks of immunogenicity and insertional mutations, simplifying the production process and reducing costs. The scalability and rapid optimization ability of LNP position them as promising candidates for CAR-T cell production. LNP technology is expected to further promote the development of CAR-T immunotherapy and provide safer and more economical treatment options.

Therefore, this paper aims to provide a comprehensive and systematic review of the application of LNP in CAR-T therapy. In this review, we initially outline the fundamental design, process, and current challenges of CAR-T therapy. Subsequently, we present the characteristics of LNP, their advantages as a gene delivery vectors, and how they improve the efficacy of CAR-T therapy.

Finally, we summarize the current research landscape of LNP applications in CAR-T therapy. This includes enhancing in vitro transfection of T cells, programming T cells in situ , facilitating T-cell activation, alleviating the side effects of CAR-T therapy, and combining CAR-T therapy with other immunotherapies. These advancements will aid in the design of mRNA delivery systems based on LNP, thereby promoting the development of CAR-T therapy.

论文信息

作者
Zhao Z、Li M、Zheng X、Gao P、Huang C、Yu Q、Jin L、Zhang L
单位
State Key Laboratory of Advanced Medical Materials and Devices, Tianjin Key Laboratory of Biomedical Materials, Key Laboratory of Biomaterials and Nanotechnology for Cancer Immunotherapy, Institute of Biomedical Engineering, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300192, People's Republic of China.China
文献类型
系统综述
期刊
Nanotechnology2025 May 16
原文标识
PubMed 40376884 · DOI 10.1088/1361-6528/add482