CAR-T（CAR-T）细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
体内 CAR-T 细胞工程：设计原则与开放问题
英文原题：In vivo CAR T cell engineering: design principles and open questions.
查看英文原题
In vivo CAR T cell engineering: design principles and open questions.
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中文摘要
嵌合抗原受体（CAR）T细胞疗法已经改变了血液系统恶性肿瘤的治疗格局，催生了七款获FDA批准的产品，但其广泛应用仍受限于高昂且复杂的生产与给药流程。目前已出现多种直接在患者体内重编程T细胞的方法，有望同时提高效力、降低成本并扩大可及性。尽管早期临床研究已证明其安全性和治疗活性，但在长期疗效、递送方式与宿主生物学之间的相互作用，以及每种递送方式的最佳疾病适应症等方面，仍存在关键问题。本综述总结了用于体内CAR-T 工程化的最先进病毒载体和非病毒载体方法的临床前与临床数据，讨论了其机遇与局限，并重点指出了将塑造这一新兴领域成熟过程的关键未解问题。
展开英文摘要原文
Chimeric antigen receptor (CAR) T cell therapy has transformed the treatment of hematologic malignancies, leading to seven FDA-approved products, but widespread adoption has been limited by costly and complex manufacturing and administration. Several methods have emerged to directly reprogram T cells within patients, with the potential to simultaneously improve potency, reduce costs, and broaden accessibility.
While early clinical studies demonstrate safety and therapeutic activity, critical questions remain regarding long-term efficacy, the interplay between means of delivery and host biology, and optimal disease indications for each delivery approach. This review summarizes the preclinical and clinical data for state-of-the-art viral and nonviral approaches for in vivo CAR-T engineering, discusses their opportunities and limitations, and highlights key open questions that will shape the maturation of this emerging field.
论文信息
- 作者
- Aalipour A、Barreiro A、Garmilla A、Birnbaum ME
- 第一作者单位
- Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA; Koch Institute for Integrative Cancer Research, Cambridge, MA, USA.United States
- 通讯作者单位
- Koch Institute for Integrative Cancer Research, Cambridge, MA, USA; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; Singapore-MIT Alliance for Research and Technology Centre, Singapore, Singapore; Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA, USA. Electronic address: mbirnb@mit.edu.United Kingdom
- 文献类型
- 综述
- 期刊
- Trends in cancer2026 May
- 原文标识
- PubMed 41887986 · DOI 10.1016/j.trecan.2026.01.007