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肝细胞通过亚亚糖蛋白受体向肝细胞

Fabricio Ramírez-Cortés¹, Petra Ménová¹

¹University of Chemistry and Technology, Prague Technická 5 16628 Prague 6 Czech Republic petra.menova@vscht.cz.

RSC medicinal chemistry

|December 4, 2024

概括

亚洲葡萄糖蛋白受体 (ASGPR) 向对肝脏特异性治疗有希望. 本综述涵盖ASGPR连接体,药物输送策略以及siRNA和ASO等核酸疗法的临床进展.

科学领域:

肝病学和药物输送
分子疗法和有针对性的输送

背景情况:

亚亚糖蛋白受体 (ASGPR) 是肝脏特异性药物输送的关键标.
了解ASGPR配体对于开发有效的肝脏向疗法至关重要.

研究的目的:

对肝脏特异性治疗的ASGPR介导向策略进行审查.
分析ASGPR向在提供核酸治疗药物的应用.
讨论RNA干扰 (RNAi) 和反感性寡核酸 (ASO) 疗法的临床发展.

主要方法:

对天然和合成的ASGPR配体进行全面的审查.
使用ASGPR的药物输送系统的分析.
深入研究临床开发中的RNA干扰 (RNAi) 和反感性寡核酸 (ASO) 疗法.

主要成果:

天然和合成的ASGPR配体是肝脏向策略的核心.
在使用ASGPR提供核酸疗法,包括siRNA和ASO方面取得了重大进展.
几种基于RNAi和ASO的疗法已获得批准或处于先进的临床试验阶段 (1-3期).

结论:

以ASGPR为媒介的向是一种强大的方法,可以推进肝脏特异性治疗.
该综述详细概述了当前ASGPR向的结合物及其治疗潜力.
未来的方向包括通过ASGPR.通过细胞外蛋白的向蛋白质降解.

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