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修饰免疫细胞的构成性IL-1RA生产可以防止IL-1介导的炎症性疾病

Mariasilvia Colantuoni^1,2, Raisa Jofra Hernandez¹, Emanuela Pettinato¹

¹San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Science translational medicine

|May 31, 2023

概括

使用造血干细胞/原始细胞 (HSPCs) 的基因疗法可以提供互白素-1受体对手 (IL-1RA) 来治疗慢性炎症. 这种方法为由IL-1驱动的免疫相关疾病提供了有希望的长期治疗方法.

科学领域:

免疫学免疫学免疫学
基因治疗基因治疗
血液学血液学血液学

背景情况:

介素-1 (IL-1) 途径的失调会导致免疫疾病和慢性炎症.
目前的IL-1阻断治疗提供症状缓解,但缺乏长期疗效.
对IL-1驱动的炎症状况需要新的治疗策略.

研究的目的:

为持续的IL-1受体抗剂 (IL-1RA) 生产开发和评估一种lentivirus (LV) 介导的基因转移策略.
利用自身造血干细胞/原始细胞 (HSPC) 进行系统性IL-1RA输送.
评估HSPC介导IL-1RA在IL-1依赖性炎症疾病中的治疗潜力.

主要方法:

开发了一种LV介导的基因转移系统,用IL-1RA基因转导小鼠和人类的HSPCs.
移植的转化HSPCs和确认稳定的IL-1RA生产和HSPC功能在体内.
评估了这种细胞介导基因疗法的有效性,在痛风的小鼠模型,冷素相关的周期性综合征和实验性自身免疫脑膜炎.

主要成果:

转化后的HSPCs稳定地产生IL-1RA,同时保持它们的干细胞特性.
通过HSPC介导的IL-1RA递送有效地减少了痛风,周期性发烧综合征和多发性硬化症模型中的炎症.
治疗成功地阻碍了中性粒细胞的招募和全身炎症.

结论:

通过HSPC介导的IL-1RA输送是IL-1驱动炎症的可行的治疗策略.
这种基因治疗方法可持续抑制组织和器官炎症.
该方法有可能用于治疗涉及IL-1通路的各种免疫相关疾病.

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