• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利:CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法,用中文像聊天一样搜索,搜遍4000万医学文献。AI智能推荐,让科研检索更轻松。

立即免费搜索

文件翻译

保留排版,准确专业,支持PDF/Word/PPT等文件格式,支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述,25分钟生成高质量综述,智能提取关键信息,辅助科研写作。

立即免费体验

下一代多重编辑的嵌合抗原受体自然杀伤细胞(CAR-NK细胞):更多编辑,更强效力?

Next-generation multiplex-edited CAR-NK cells: more edits, more power?

作者信息

Bexte Tobias, Wagner Dimitrios L

机构信息

Department of Pediatrics, Experimental Immunology and Celltherapy, Goethe University Frankfurt, Frankfurt am Main, Germany

Institute for Transfusion Medicine and Immunohematology, German Red Cross Hesse, Frankfurt am Main, HE, Germany.

出版信息

J Immunother Cancer. 2025 Sep 15;13(9):e012841. doi: 10.1136/jitc-2025-012841.

DOI:10.1136/jitc-2025-012841
PMID:40954078
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12439146/
Abstract

First clinical trials demonstrated the safety of adoptive cell transfer with allogeneic natural killer (NK) cell products from healthy donors, making them an attractive candidate for 'off-the-shelf' chimeric antigen receptor (CAR)-immune cell therapy. However, reduced persistence and inactivation of NK cells by immunosuppressive cues likely limit the performance of CAR-redirected NK cells. Wang and colleagues demonstrate that multiplex CRISPR base editing allows optimization of the intrinsic functionality of CAR-NK cells improving their therapeutic potential. In contrast to conventional CRISPR-Cas nucleases, base editing avoided most double-stranded DNA breaks while enabling highly efficient editing at up to six sites simultaneously. The study further demonstrates the feasibility of a non-viral approach to integrate CAR transgene and multiplex base editing of several immune checkpoints in NK cells using a single electroporation. CAR-NK cells harboring up to three base edits demonstrate improved potency over unedited counterparts in vitro. Xenograft mouse models confirmed increased potency, but also indicated signs of organ toxicity - a phenomenon that will require future studies prior to clinical translation. The study demonstrates that CRISPR base editing is a powerful tool to unleash the full cytotoxic potential of NK cells, but it also warrants the question: How many internal breaks can be removed without hurting CAR-NK cell therapy's impeccable safety record?

摘要

首次临床试验证明了使用来自健康供体的同种异体自然杀伤(NK)细胞产品进行过继性细胞转移的安全性,使其成为“现成可用”的嵌合抗原受体(CAR)免疫细胞疗法的一个有吸引力的候选者。然而,免疫抑制信号导致NK细胞的持久性降低和失活,这可能会限制CAR重定向NK细胞的性能。王及其同事证明,多重CRISPR碱基编辑能够优化CAR-NK细胞的内在功能,提高其治疗潜力。与传统的CRISPR-Cas核酸酶不同,碱基编辑避免了大多数双链DNA断裂,同时能够在多达六个位点同时进行高效编辑。该研究进一步证明了一种非病毒方法的可行性,即通过单次电穿孔将CAR转基因整合到NK细胞中,并对多个免疫检查点进行多重碱基编辑。携带多达三个碱基编辑的CAR-NK细胞在体外表现出比未编辑的同类细胞更高的效力。异种移植小鼠模型证实了效力的提高,但也显示出器官毒性的迹象——这一现象在临床转化之前需要未来的研究。该研究表明,CRISPR碱基编辑是释放NK细胞全部细胞毒性潜力的有力工具,但它也引发了一个问题:在不损害CAR-NK细胞疗法无可挑剔的安全记录的情况下,可以消除多少内部断裂?

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/762a/12439146/0050ea639b03/jitc-13-9-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/762a/12439146/0050ea639b03/jitc-13-9-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/762a/12439146/0050ea639b03/jitc-13-9-g001.jpg

相似文献

1
Next-generation multiplex-edited CAR-NK cells: more edits, more power?下一代多重编辑的嵌合抗原受体自然杀伤细胞(CAR-NK细胞):更多编辑,更强效力?
J Immunother Cancer. 2025 Sep 15;13(9):e012841. doi: 10.1136/jitc-2025-012841.
2
Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells.将嵌合抗原受体无病毒CRISPR敲入KLRC1可产生高效的GD2特异性自然杀伤细胞。
Mol Ther. 2025 Mar 5;33(3):1014-1030. doi: 10.1016/j.ymthe.2025.01.024. Epub 2025 Jan 14.
3
Allogeneic CAR-engineered cellular therapy for relapsed and refractory large B cell lymphoma: a systematic review and meta-analysis.异基因嵌合抗原受体工程化细胞疗法治疗复发难治性大B细胞淋巴瘤:一项系统评价和荟萃分析。
Front Immunol. 2025 Jul 8;16:1585556. doi: 10.3389/fimmu.2025.1585556. eCollection 2025.
4
Overcoming Immune Barriers in Allogeneic CAR-NK Therapy: From Multiplex Gene Editing to AI-Driven Precision Design.克服异基因CAR-NK疗法中的免疫障碍:从多重基因编辑到人工智能驱动的精准设计
Biomolecules. 2025 Jun 26;15(7):935. doi: 10.3390/biom15070935.
5
Precision targeting of rhabdomyosarcoma by combining primary CAR NK cells and radiotherapy.通过联合原发性嵌合抗原受体自然杀伤细胞(CAR NK细胞)和放射疗法精准靶向横纹肌肉瘤。
J Immunother Cancer. 2025 Jul 7;13(7):e011330. doi: 10.1136/jitc-2024-011330.
6
Multiplex engineering using microRNA-mediated gene silencing in CAR T cells.在嵌合抗原受体(CAR)T细胞中使用微小RNA介导的基因沉默进行多重工程。
Front Immunol. 2025 Aug 21;16:1647433. doi: 10.3389/fimmu.2025.1647433. eCollection 2025.
7
Tracing the development of CAR-T cell design: from concept to next-generation platforms.追溯嵌合抗原受体T细胞(CAR-T)设计的发展:从概念到下一代平台。
Front Immunol. 2025 Jul 17;16:1615212. doi: 10.3389/fimmu.2025.1615212. eCollection 2025.
8
CAR-NK, a Splendid Strategy for Cancer, Especially for Gynecologic Tumor.嵌合抗原受体自然杀伤细胞疗法(CAR-NK),一种治疗癌症的卓越策略,尤其适用于妇科肿瘤。
Immun Inflamm Dis. 2025 Jun;13(6):e70210. doi: 10.1002/iid3.70210.
9
Constitutive IL-7 signaling promotes CAR-NK cell survival in the solid tumor microenvironment but impairs tumor control.组成性白细胞介素-7信号通路促进实体瘤微环境中嵌合抗原受体自然杀伤(CAR-NK)细胞的存活,但损害肿瘤控制。
J Immunother Cancer. 2025 Jul 23;13(7):e010672. doi: 10.1136/jitc-2024-010672.
10
In vitro machine learning-based CAR T immunological synapse quality measurements correlate with patient clinical outcomes.基于体外机器学习的 CAR T 免疫突触质量测量与患者临床结果相关。
PLoS Comput Biol. 2022 Mar 18;18(3):e1009883. doi: 10.1371/journal.pcbi.1009883. eCollection 2022 Mar.

本文引用的文献

1
Precision enhancement of CAR-NK cells through non-viral engineering and highly multiplexed base editing.通过非病毒工程和高度多重碱基编辑提高CAR-NK细胞的精准度。
J Immunother Cancer. 2025 May 7;13(5):e009560. doi: 10.1136/jitc-2024-009560.
2
Interleukin-21 engineering enhances NK cell activity against glioblastoma via CEBPD.白细胞介素-21 工程通过 CEBPD 增强 NK 细胞对神经胶质瘤的活性。
Cancer Cell. 2024 Aug 12;42(8):1450-1466.e11. doi: 10.1016/j.ccell.2024.07.007.
3
Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors.
使用最小化 DNA 载体的非病毒睡眠美人转座子工程化有效的 CAR NK 细胞。
Mol Ther. 2024 Jul 3;32(7):2357-2372. doi: 10.1016/j.ymthe.2024.05.022. Epub 2024 May 14.
4
Integration of ζ-deficient CARs into the CD3ζ gene conveys potent cytotoxicity in T and NK cells.ζ 缺陷型嵌合抗原受体整合到 CD3ζ 基因中可赋予 T 和 NK 细胞强大的细胞毒性。
Blood. 2024 Jun 20;143(25):2599-2611. doi: 10.1182/blood.2023020973.
5
Natural killer cell therapies.自然杀伤细胞疗法。
Nature. 2024 Feb;626(8000):727-736. doi: 10.1038/s41586-023-06945-1. Epub 2024 Feb 21.
6
Safety, efficacy and determinants of response of allogeneic CD19-specific CAR-NK cells in CD19 B cell tumors: a phase 1/2 trial.同种异体 CD19 特异性 CAR-NK 细胞治疗 CD19 B 细胞肿瘤的安全性、有效性和反应决定因素:一项 1/2 期试验。
Nat Med. 2024 Mar;30(3):772-784. doi: 10.1038/s41591-023-02785-8. Epub 2024 Jan 18.
7
Nonviral technologies can pave the way for CAR-NK cell therapy.非病毒技术可以为 CAR-NK 细胞疗法铺平道路。
J Leukoc Biol. 2023 Oct 26;114(5):475-486. doi: 10.1093/jleuko/qiad074.
8
Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing.评估和推进 CRISPR-Cas 工具的安全性:从 DNA 到 RNA 编辑。
Nat Commun. 2023 Jan 13;14(1):212. doi: 10.1038/s41467-023-35886-6.
9
Engineering CAR-NK cells to secrete IL-15 sustains their anti-AML functionality but is associated with systemic toxicities.工程化 CAR-NK 细胞分泌 IL-15 可维持其抗 AML 功能,但与全身毒性有关。
J Immunother Cancer. 2021 Dec;9(12). doi: 10.1136/jitc-2021-003894.
10
CRISPR-Cas9 cytidine and adenosine base editing of splice-sites mediates highly-efficient disruption of proteins in primary and immortalized cells.CRISPR-Cas9 胞嘧啶和腺嘌呤碱基编辑剪接位点可介导在原代和永生化细胞中高效破坏蛋白质。
Nat Commun. 2021 Apr 23;12(1):2437. doi: 10.1038/s41467-021-22009-2.