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在体内将基因传递给免疫细胞

Jamison C Siebart¹, Ching S Chan¹, Xinyi Yao¹

¹The Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA 30332, USA.

Current opinion in biotechnology

|July 7, 2024

概括

在体内基因输送车辆为克服制造挑战和与当前免疫细胞疗法相关的不良影响提供了一个有希望的解决方案. 这种方法旨在民主化各种疾病的细胞免疫疗法.

科学领域:

免疫学免疫学免疫学
基因治疗基因治疗
细胞疗法细胞疗法

背景情况:

免疫细胞疗法,或"活药",利用治疗性转基因来修改免疫细胞功能.
目前的自身治疗方法面临的局限性包括高成本,漫长的制造时间和与免疫相关的显著不良影响.

研究的目的:

探索新的基因传递载体,以在体内准免疫细胞.
解决ex vivo制造的局限性,并改善治疗结果.
为了降低毒性和提高细胞免疫疗法的可用性.

主要方法:

开发多样化的基因传递载体.
在体内向特定的免疫细胞子集.
对各种疾病进行临床前和临床评估.

主要成果:

新兴的基因输送载体显示出在体内免疫细胞修饰的潜力.
在现场调节治疗反应和降低毒性是关键目标.
成功的体内基因传递对于推进细胞免疫疗法至关重要.

结论:

在体内基因传递代表了细胞免疫疗法的范式转变.
克服制造和毒性障碍对于更广泛的患者接入至关重要.
这项技术对于先进的基于免疫的治疗方法的民主化至关重要.

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