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体内 CAR 细胞治疗:从实验室到临床

英文原题:In vivo CAR cell therapy: from bench to bedside.

查看英文原题

In vivo CAR cell therapy: from bench to bedside.

PubMed 2025/11/20(内容时间) J Hematol Oncol Q1 · IF 47.8(JCR 2025)

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中文摘要

CAR-T 细胞疗法近年来已成为治疗难治性和复发性血液系统恶性肿瘤的高效手段。然而,自体ex vivo CAR-T 细胞相关的复杂制备流程、严格的物流要求以及漫长的生产周期,使其成本高昂且许多患者难以获得。相比之下,in vivo CAR-T 疗法直接将编码CAR的转基因递送至内源性T细胞,在原位对其进行重编程。该方法免除了传统CAR-T 疗法中固有的白细胞分离术、ex vivo细胞制备和淋巴细胞清除性化疗。因此,in vivo CAR-T 代表了一种更高效、更经济的范式,将CAR-T 从个体化细胞产品转变为真正的“即用型”治疗药物。本综述总结了in vivo CAR细胞疗法从基础到临床的最新研究进展,为推进其临床转化提供思路。

展开英文摘要原文

Chimeric antigen receptor T cell (CAR-T) therapy has emerged as a highly efficacious treatment for refractory and relapsed hematological malignancies in recent years.

However, the complex manufacturing procedures, stringent logistical requirements, and protracted production timelines associated with autologous ex vivo CAR-T cells render them costly and inaccessible to many patients. In contrast, in vivo CAR-T therapy directly delivers CAR-encoding transgenes to endogenous T cells, reprogramming them in situ. This approach obviates the need for apheresis, ex vivo cell manufacturing, and lymphodepleting chemotherapy inherent in conventional CAR-T therapy.

Consequently, in vivo CAR-T represents a more efficient and economical paradigm, transforming CAR-T from individualized cellular products towards truly "ready-to-use" therapeutics. This review summarizes the latest research progress in in vivo CAR cell therapies, spanning from bench to bedside, to provide insights for advancing their clinical translation.

论文信息

作者
Xu J、Chen Z、Su L、Ren A、Mei H
第一作者单位
Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, 1277 Jiefang Avenue, Wuhan, 430022, China.China
通讯作者单位
Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, 1277 Jiefang Avenue, Wuhan, 430022, China. hmei@hust.edu.cn.China
文献类型
综述 · 非美国政府资助研究
期刊
Journal of hematology & oncology2025 Nov 20
原文标识
PubMed 41261423 · DOI 10.1186/s13045-025-01759-2