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通过3'终端结合改善叶酸结合完全化学修饰siRNAs的细胞积累

Keiichi Motosawa¹, Junko Iwano¹, Toshimasa Harumoto¹

¹Research Unit, Research Division, Kyowa Kirin Co., Ltd., 3-6-6 Asahi-machi, Machida-shi, Tokyo 194-8533, Japan.

|August 11, 2025

概括

叶酸 (FA) 结合增强了小干扰RNA (siRNA) 向叶酸受体1 (FOLR1) 表达细胞的传递. 在反意义链上修改FA-siRNA.

科学领域:

生物医学工程生物医学工程
分子生物学分子生物学
药物输送系统药物输送系统

背景情况:

叶酸 (FA) 结合是一种经过验证的瘤特异性小分子输送方法.
使用FA记录了微RNA和小干扰RNA (siRNA) 等寡核酸的向输送.
作为一种常见的临床平台,FA结合的化学修饰siRNA的疗效需要进一步研究.

研究的目的:

使用FA结合来增强siRNA的细胞积累和基因淘汰 (KD).
设计和评估各种FA-siRNA格式,以提高传递和疗效.
研究化学修饰对FOLR1表达细胞中FA-siRNA性能的影响.

主要方法:

各种叶酸结合小干扰RNA (FA-siRNA) 格式的设计.
在叶酸受体1 (FOLR1) 表达细胞中的细胞积累和基因淘汰 (KD) 的评估.
通过3'端反意义链结合对siRNA稳定性的评估.

主要成果:

在FA-siRNA增强细胞内积累的反意义链的3'端的替代物的结合.
这种修改可能会稳定siRNA结构.
开发的FA-siRNA格式在FOLR1表达细胞中显示出有希望的基因沉默能力.

结论:

这项研究提出了一种新的方法来增强表达叶酸受体1 (FOLR1) 的癌细胞中的基因沉默.
修改FA-siRNA,特别是在反意义链上,可以改善细胞吸收和稳定性.
这一策略为FOLR1阳性瘤的向基因治疗提供了一个有希望的平台.

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