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通过eVLP介导的Cas9交付,以防止IBMIR在岛屿移植中的发生

Manju Shrestha^1,2, Yeonji Kim³, Subin Park¹

¹Department of Precision Medicine, Sungkyunkwan University School of Medicine, Suwon, 16419, Republic of Korea.

Small (Weinheim an der Bergstrasse, Germany)

|November 18, 2025

概括

从小岛中去除组织因子 (TF) 和等离子体激活剂抑制剂-1 (PAI-1) 的基因编辑可以防止即时的血液介导炎症反应 (IBMIR). 这通过减少炎症和移植损失,改善了1型糖尿病的岛屿移植结果.

科学领域:

免疫学免疫学免疫学
内分泌学在内分泌学.
基因治疗基因治疗

背景情况:

岛屿移植通过替代丢失的胰岛素产生β细胞,为1型糖尿病提供了潜在的治疗方法.
即时血液介导炎症反应 (IBMIR) 是一个主要的障碍,由于血液接触,导致移植小岛的快速破坏.
组织因子 (TF) 和等离子体激活剂抑制剂-1 (PAI-1) 是IBMIR的关键介质,分别启动凝血和抑制纤维解质.

研究的目的:

通过淘汰TF和PAI-1基因以减轻IBMIR,对老鼠小岛进行基因改造.
评估TF和PAI-1基因淘汰对岛屿活力,功能和IBMIR抑制的影响.
在1型糖尿病的小鼠模型中评估修饰小岛的治疗效果.

主要方法:

利用工程病毒样粒子 (eVLP) 介导的CRISPR-Cas9系统,在小鼠群岛中针对TF和PAI-1的向基因淘汰.
证实有效的基因下调,没有目标外突变,并评估了小岛的活力和功能.
将移植的修改小岛移植到链毒素诱导的糖尿病小鼠中,并监测血糖控制和IBMIR标志物.

主要成果:

成功下调小岛的TF和PAI-1表达,而不会损害它们的生存能力或功能.
移植的缺少TF和PAI-1的小岛在糖尿病小鼠中显著改善了血糖控制.
在接受者小鼠中降低了氨酸-抗氨酸复合物和补充成分3a的水平,证实了IBMIR抑制.

结论:

在小岛中对TF和PAI-1进行基因编辑是防止IBMIR的可行策略.
这种方法增强了小岛移植对1型糖尿病的治疗潜力.
针对TF和PAI-1提供了一条有前途的途径,可以改善移植的存活率和长期的移植成功.

关键词:

即时的血液介导的炎症反应.岛屿移植移植小岛移植塑原激活剂抑制剂-1-1 组织因子组织因子类似病毒的颗粒.

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