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细胞治疗革命:体内 CAR-T 细胞治疗的突破与未来前景

英文原题:Revolution in Cell Therapy: In Vivo Chimeric-Antigen-Receptor-T-Cell Therapy Breakthroughs and Promises for the Future.

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Revolution in Cell Therapy: In Vivo Chimeric-Antigen-Receptor-T-Cell Therapy Breakthroughs and Promises for the Future.

PubMed 2025/10/09(内容时间) Research (Wash D C) Q1 · IF 12.9(JCR 2025)

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中文摘要

嵌合抗原受体(CAR)T细胞疗法治疗血液系统肿瘤取得重要成果,但传统CAR-T 疗法存在体外制备流程复杂、成本高、患者T细胞功能较低、难以多次给药以及实体瘤疗效有限等问题,因此亟需发展体内CAR-T 疗法。该方法通过递送系统将CAR基因组分直接送入宿主体内T细胞,实现原位重编程,避免体外操作,在治疗时效性、经济可行性和疗效持久性方面具有重要优势。本文介绍体内CAR-T 治疗研究现状,包括递送系统开发,以及CAR-T 疗法在血液系统恶性肿瘤、实体瘤、自身免疫性疾病和感染性疾病中的应用;并讨论高效递送、安全调控、持久性和功能优化,以及克服肿瘤微环境等问题。文章还探讨具有前景的创新解决方案,尤其关注体内CAR-T 疗法的技术突破、适应症拓展和产业化发展。

展开英文摘要原文

Chimeric-antigen-receptor (CAR)-T-cell therapy has achieved important results in the treatment of hematological tumors, but traditional CAR-T-cell therapy has the problems of complicated in vitro preparation processes, high cost, low T-cell function in patients, difficulty in multiple dosing, and limited treatment efficacy in solid tumors. In vivo CAR-T-cell therapy has emerged as needed. The CAR gene component is directly delivered to T cells in the host through the delivery system to achieve in situ reprogramming, avoids in vitro manipulation, and has important advantages in terms of the timeliness of treatment, economic feasibility, and persistence of treatment.

This paper describes the current state of research on in vivo CAR-T-cell therapy, including the development of delivery systems and the application of CAR-T-cell therapy in treating hematological malignancies, solid tumors, autoimmune diseases, and infectious diseases, as well as discussions on efficient delivery, safety regulation, persistence and functional optimization, and overcoming the tumor microenvironment.

It also explores innovative solutions, which hold promise for the future development of in vivo CAR-T-cell therapy, particularly in terms of technological breakthroughs, expansion of treatment indications, and industrialization.

论文信息

作者
Lai M、Shao W、Mao J、Ye Q
第一作者单位
Department of Nephrology, Children's Hospital, and Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou 310052, China.China
通讯作者单位
Department of Laboratory Medicine, Children's Hospital, and Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou 310052, China.China
文献类型
综述
期刊
Research (Washington, D.C.)2025
原文标识
PubMed 41079670 · DOI 10.34133/research.0917