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口服基因递送平台基于糖醇基托-PEG-乳酸联合体

Seonmi Jang¹, Lia Priscilla², Chang Woo Lee¹

¹Department of Bioengineering, College of Engineering, Hanyang University, Seoul 04763, Republic of Korea.

Journal of controlled release : official journal of the Controlled Release Society

|November 28, 2025

概括

使用甘醇基托的新型口服基因传递系统有效地传递纤维细胞生长因子21 (FGF21) 用于2型糖尿病治疗. 这种方法显著改善了小鼠的葡萄糖和胰岛素敏感性.

科学领域:

生物技术是生物技术.
基因治疗基因治疗
代谢障碍代谢障碍代谢障碍

背景情况:

腹腔注射是代谢障碍的标准,但口服基因输送面临着诸如酶降解和吸收不良等挑战.
2型糖尿病 (T2D) 管理需要有效的治疗策略来改善葡萄糖和脂质代谢.

研究的目的:

开发一种针对代谢障碍的非病毒性口服基因传递系统.
通过使用一种新型平台,评估口服输送纤维细胞生长因子21 (FGF21) 的疗效.

主要方法:

开发了一种用于基因传递的甘基托-马利胺-聚乙烯甘-N-氧苏胺-乳酸 (GPL) 平台.
利用乳二烯受体介导的细胞转移来增强FGF21的肠道吸收.
给高脂肪饮食诱导的T2D小鼠口服GPL/FGF21多重复合物.

主要成果:

通过口服,可以达到与腹腔内注射相比的循环FGF21水平.
在T2D小鼠中,食葡萄糖水平降低了~37%.
与全身FGF21表达的胰岛素敏感性有~47%的改善.

结论:

基于GPL的口服基因输送系统是对代谢疾病的有希望的策略.
这个平台克服了口服基因传递的局限性,为亲肠道输送提供了替代方案.
该系统在治疗各种代谢障碍方面具有广泛的适用性.

关键词:

纤维细胞生长因子21 (FGF21)基因治疗是一种基因疗法.糖醇基托-马利胺基-聚乙烯糖醇-N-氧苏胺基-乳酸铁素 (GPL)这是口头传递.二型糖尿病 (T2D) 是一种糖尿病.

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