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.
CAR-T（CAR-T）细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
直接体内 CAR-T 细胞工程化
英文原题：Direct in vivo CAR T cell engineering.
查看英文原题
Direct in vivo CAR T cell engineering.
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中文摘要
经工程化改造表达精心设计嵌合抗原受体（CAR）的T细胞，是治疗复发/难治性血液癌症的强效药物，并有潜力革新许多其他疾病的疗法。为避免大规模实施体外制备过继细胞疗法产品所涉及的复杂流程和高昂费用，过去数年中，直接输注纳米颗粒制剂核酸或正在开发的工程化病毒载体，以在体内生成CAR-T 细胞等替代策略备受关注。本文以体外制备流程为参照框架，概述直接体内生成策略，并讨论临床前模型的新兴数据，突出体内方法的效力、对新疾病适应证的适用性，以及临床应用准备阶段仍面临的挑战，包括递送特异性、长期疗效和安全性。
展开英文摘要原文
T cells modified to express intelligently designed chimeric antigen receptors (CARs) are exceptionally powerful therapeutic agents for relapsed and refractory blood cancers and have the potential to revolutionize therapy for many other diseases.
To circumvent the complexity and cost associated with broad-scale implementation of ex vivo manufactured adoptive cell therapy products, alternative strategies to generate CAR T cells in vivo by direct infusion of nanoparticle-formulated nucleic acids or engineered viral vectors under development have received a great deal of attention in the past few years.
Here, we outline the ex vivo manufacturing process as a motivating framework for direct in vivo strategies and discuss emerging data from preclinical models to highlight the potency of the in vivo approach, the applicability for new disease indications, and the remaining challenges associated with clinical readiness, including delivery specificity, long term efficacy, and safety.
论文信息
- 作者
- Short L、Holt RA、Cullis PR、Evgin L
- 第一作者单位
- Michael Smith Genome Sciences Department, BC Cancer Research Institute, Vancouver, BC, Canada; Interdisciplinary Oncology Program, University of British Columbia, Vancouver, BC, Canada.Canada
- 通讯作者单位
- Michael Smith Genome Sciences Department, BC Cancer Research Institute, Vancouver, BC, Canada; Interdisciplinary Oncology Program, University of British Columbia, Vancouver, BC, Canada; Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada. Electronic address: levgin@bcgsc.ca.Canada
- 文献类型
- 综述 · 非美国政府资助研究
- 期刊
- Trends in pharmacological sciences2024 May
- 原文标识
- PubMed 38614815 · DOI 10.1016/j.tips.2024.03.004