嵌合抗原受体
离体
体内
细胞疗法
医学
免疫疗法
免疫学
计算机科学
计算生物学
干细胞
生物
免疫系统
生物技术
细胞生物学
作者
Lauralie Short,Robert A. Holt,Pieter R. Cullis,Laura Evgin
标识
DOI:10.1016/j.tips.2024.03.004
摘要
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.
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