强化植物衍生的脂质纳米颗粒用于口服精确的表观基因组编辑在结肠疾病
Qiang Gao1,2, Yunxia Gao3, Yingui Cao1
1State Key Laboratory of Resource Insects, College of Sericulture, Textile, and Biomass Sciences, Southwest University, Beibei, Chongqing 400715, China.
Science advances
|September 26, 2025
概括
这项研究引入了一个新的口服基因组编辑平台,使用脂质纳米粒子 (LNP) 进行精确的CRISPR-Cas9传递. 该系统有效地向巨细胞中的Lsd1,减轻结肠炎症和瘤发生,用于潜在的结肠直肠疾病治疗.
科学领域:
- 生物技术是生物技术.
- 分子生物学分子生物学
- 基因组学就是基因组学.
背景情况:
- 基因编辑CRISPR-Cas9面临的交付挑战,特别是口服.
- 氨酸特异性去甲基酶1 (Lsd1) 与结肠炎症和癌症发展有关.
研究的目的:
- 开发一个口腔基因组编辑平台,以准结肠中的Lsd1.
- 为了提高CRISPR-Cas9的传递和治疗结肠炎和相关瘤发生的疗效.
主要方法:
- 开发TPGS-RNP@LNP,一个口服平台,用TPGS将Lsd1向性核核蛋白 (RNP) 封装在脂质纳米粒子 (LNP) 中.
- 利用银河糖受体介导的内细胞分裂来向巨细胞并确保RNP的核传递.
- 在大肠炎模型中评估Lsd1编辑效率,表观遗传调制 (H3K4甲基化) 和治疗效果.
主要成果:
- 与商业CRISPRMAX (43.0%) 相比,TPGS-RNP@LNPs表现出更高的Lsd1编辑效率 (59.7%).
- 口服调节了表观遗传状态,减少了炎症,恢复了上皮屏障,并抑制了结肠炎相关的瘤发生.
- 该平台显示增强了胃肠道稳定性和结肠粘液透.
结论:
- TPGS-RNP@LNPs代表了一个有前途的基于LNP的口服RNP输送系统,用于精确的结直肠疾病治疗.
- 这个平台克服了CRISPR-Cas9应用在治疗结肠炎症和癌症条件的口服交付挑战.
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