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在体内化学抗原受体 (CAR) -T细胞疗法

Adrian Bot¹, Andrew Scharenberg², Kevin Friedman³

¹Capstan Therapeutics, San Diego, CA, USA. abot@capstantx.com.

Nature reviews. Drug discovery

|September 30, 2025

概括

在体内的CAR-T细胞工程可以在体内产生CAR-T细胞,克服传统疗法的制造限制. 这种方法对治疗血液癌症和自身免疫性疾病充满希望.

科学领域:

免疫治疗是一种免疫疗法.
细胞疗法细胞疗法
基因工程是一种基因工程.

背景情况:

化学抗原受体 (CAR) -T细胞疗法已经彻底改变了血液癌症治疗.
目前的ex vivoCAR-T细胞疗法在制造,可扩展性和临床一致性方面面临挑战.

研究的目的:

审查体内CAR-T细胞工程中的进步.
探索能够在体内产生CAR-T细胞的技术.
讨论体内方法在更广泛的治疗应用中的潜力.

主要方法:

使用向传递系统,如晶状病毒载体和脂质纳米粒子.
将CAR编码的遗传物质引入患者内部的内源性T细胞.
利用病毒学,RNA药物和纳米技术的进步.

主要成果:

早期的临床研究表明有效的T细胞转导和持续的CAR表达.
对抗瘤活性的初步证据建立了 in vivo CAR-T 生产的概念证明.
在体内工程消除了ex vivo细胞处理和调节的需要.

结论:

在体内CAR-T细胞工程提供了一个可扩展和潜在的更有效的替代ex vivo方法.
这一策略可以扩大CAR-T细胞治疗的应用范围,不仅仅是血液性恶性瘤,还包括自身免疫性疾病.
技术进步正在推动开发更安全,更广泛适用的体内CAR-T细胞疗法.

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