无抗体修饰的心磷脂模拟脂质纳米颗粒提供抗衰老体内CAR-T治疗炎症衰老。

IF 11.7 1区 医学 Q1 CELL BIOLOGY
Cell Reports Medicine Pub Date : 2025-07-15 Epub Date: 2025-07-01 DOI:10.1016/j.xcrm.2025.102209
Zihan Zhang, Bin Ma, Buyao Li, Zhiwei Li, Min Gao, Hailong Zhao, Rui Peng, Jiang Hu, Yu Wang, Wei You, Xun Gui, Rui Wang, Xiaoqing Hu, Beidi Chen, Yuanjie Zhang, Yanyun Hao, Xiaolin Sun, Peishi Rao, Liang Zhang, Ming Lu, Demin Zhou, Yun Yang, Mi Deng, Lei Miao
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引用次数: 0

摘要

基于mrna的体内嵌合抗原受体(CAR)-T细胞工程提供了优于体外治疗的优势,包括流线型制造和瞬时表达。然而,目前的运输方法需要抗体修饰的车辆,这在制造上存在挑战。在这项研究中,受到心磷脂的启发,我们在体外和体内鉴定了心磷脂样二磷酰胺脂质,它可以改善T细胞转染,而不靶向配体。这种对T细胞有利的趋向性可能是由于脂质的堆积、形状和刚性。包封环状RNA进一步延长了脾脏和T细胞中mRNA的表达。利用PL40脂质纳米颗粒,我们递送了一种编码CAR的mRNA,靶向衰老和炎症抗原尿激酶型纤溶酶原激活物受体(uPAR),减轻uPAR相关的肝纤维化和类风湿性关节炎(RA)。人类单细胞测序证实了uPAR与RA的衰老和炎症相关。为了促进临床翻译,我们筛选并人源化了针对uPAR的单链可变片段(scFvs),建立了一种PL40 mrna编码的人源化uPAR CAR,具有治疗衰老炎症性疾病的潜力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Cardiolipin-mimic lipid nanoparticles without antibody modification delivered senolytic in vivo CAR-T therapy for inflamm-aging.

mRNA-based in vivo chimeric antigen receptor (CAR)-T cell engineering offers advantages over ex vivo therapies, including streamlined manufacturing and transient expression. However, current delivery methods require antibody-modified vehicles with manufacturing challenges. In this study, inspired by cardiolipin, we identify cardiolipin-like di-phosphoramide lipids that improve T cell transfection without targeting ligands, both in vitro and in vivo. The T cell-favored tropism is likely due to the lipid's packing, shape, and rigidity. Encapsulating circular RNA further prolongs mRNA expression in the spleen and T cells. Using PL40 lipid nanoparticles, we deliver mRNA encoding a CAR targeting the senolytic and inflammatory antigen urokinase-type plasminogen activator receptor (uPAR), alleviating uPAR-related liver fibrosis and rheumatoid arthritis (RA). Single-cell sequencing in humans confirms uPAR's relevance to senescence and inflammation in RA. To facilitate clinical translation, we screen and humanize single-chain variable fragments (scFvs) against uPAR, establishing a PL40 mRNA-encoded humanized uPAR CAR with potential for treating aging-inflamed disorders.

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来源期刊
Cell Reports Medicine
Cell Reports Medicine Biochemistry, Genetics and Molecular Biology-Biochemistry, Genetics and Molecular Biology (all)
CiteScore
15.00
自引率
1.40%
发文量
231
审稿时长
40 days
期刊介绍: Cell Reports Medicine is an esteemed open-access journal by Cell Press that publishes groundbreaking research in translational and clinical biomedical sciences, influencing human health and medicine. Our journal ensures wide visibility and accessibility, reaching scientists and clinicians across various medical disciplines. We publish original research that spans from intriguing human biology concepts to all aspects of clinical work. We encourage submissions that introduce innovative ideas, forging new paths in clinical research and practice. We also welcome studies that provide vital information, enhancing our understanding of current standards of care in diagnosis, treatment, and prognosis. This encompasses translational studies, clinical trials (including long-term follow-ups), genomics, biomarker discovery, and technological advancements that contribute to diagnostics, treatment, and healthcare. Additionally, studies based on vertebrate model organisms are within the scope of the journal, as long as they directly relate to human health and disease.
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