Bexte Tobias, Wagner Dimitrios L
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.
J Immunother Cancer. 2025 Sep 15;13(9):e012841. doi: 10.1136/jitc-2025-012841.
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?
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?