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Gene editing to enhance the efficacy of cancer cell therapies
Molecular Therapy ( IF 12.4 ) Pub Date : 2021-10-18 , DOI: 10.1016/j.ymthe.2021.10.001
Tara Murty 1 , Crystal L Mackall 2
Affiliation  

Adoptive T cell therapies have shown impressive signals of activity, but their clinical impact could be enhanced by technologies to increase T cell potency and diminish the cost and labor involved in manufacturing these products. Gene editing platforms are under study in this arena to (1) enhance immune cell potency by knocking out molecules that inhibit immune responses; (2) deliver genetic payloads into precise genomic locations and thereby enhance safety and/or improve the gene expression profile by leveraging physiologic promoters, enhancers, and repressors; and (3) enable off-the-shelf therapies by preventing alloreactivity and immune rejection. This review discusses gene editing approaches that have been the best studied in the context of human T cells and adoptive T cell therapies, summarizing their current status and near-term potential for translation.



中文翻译:

基因编辑增强癌细胞疗法的功效

过继性 T 细胞疗法已显示出令人印象深刻的活性信号,但可以通过提高 T 细胞效力并减少制造这些产品的成本和劳动力的技术来增强其临床影响。该领域正在研究基因编辑平台,以(1)通过敲除抑制免疫反应的分子来增强免疫细胞的效力;(2) 将遗传有效负载传递到精确的基因组位置,从而通过利用生理启动子、增强子和阻遏子来增强安全性和/或改善基因表达谱;(3) 通过防止同种异体反应和免疫排斥来实现现成疗法。这篇综述讨论了在人类 T 细胞和过继性 T 细胞疗法背景下研究得最好的基因编辑方法,总结了它们的现状和近期的转化潜力。

更新日期:2021-11-03
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