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体内 CAR 基因治疗的前景与潜在陷阱

英文原题:Promises and potential pitfalls of in vivo CAR gene therapy.

PubMed 2026/07/30(内容时间) Blood Q1 · IF 23.9(JCR 2025)

研究概要

经体外制备的表达嵌合抗原受体(CAR)的基因修饰T细胞已在淋巴系统血液恶性肿瘤患者中显示出高疗效,但其流程复杂性限制了患者的可及性。

中文摘要

经体外制备的表达嵌合抗原受体(CAR)的基因修饰T细胞已在淋巴系血液恶性肿瘤患者中显示出高疗效,但其流程复杂性限制了患者的可及性。体内方法在体内生成表达CAR的T细胞,但可能面临其自身的挑战。近期临床报告为体内CAR基因递送的可行性和治疗潜力提供了概念验证。我们总结了临床证据,并讨论了该方法的关键技术、生物学和安全性考量。

展开英文摘要原文

Genetically modified T cells expressing chimeric antigen receptors (CARs) manufactured ex vivo have demonstrated high efficacy in patients with lymphoid hematologic malignancies, but their logistic complexity limits patient access. In vivo approaches generate the CAR-expressing T cells within the body but may have their own challenges. Recent clinical reports provide proof of concept for the feasibility and therapeutic potential of in vivo CAR gene delivery. We summarize clinical evidence and discuss the critical technical, biological, and safety considerations of this approach.

论文信息

作者
Wagner DL、Elsallab M、Maus MV
第一作者单位
Center for Cell and Gene Therapy and Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX.United States
通讯作者单位
Cellular Immunotherapy Program, Massachusetts General Hospital, Mass General Brigham Cancer Institute, Boston MA.United States
文献类型
综述
期刊
Blood2026 Jul 30
原文标识
PubMed 42172553 · DOI 10.1182/blood.2025031898