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一种多功能战略,用于转化阴离子聚合物,以实现高效和器官选择性的mRNA输送

Lixin Lin¹, Kexin Su¹, Xinyue Zhang¹

¹Eye Center of the Second Affiliated Hospital of Zhejiang University School of Medicine College of Pharmaceutical Sciences, Liangzhu Laboratory, State Key Laboratory of Advanced Drug Delivery and Release Systems, Zhejiang University, Hangzhou 310058, China.

Angewandte Chemie (International ed. in English)

|February 10, 2025

概括

研究人员开发了一种多功能策略,用于制造脂和化聚合物 (PAP),用于向mRNA输送. 这些PAP显著增强体内mRNA表达,并使器官特异性输送成为可能,显示出mRNA治疗的巨大潜力.

科学领域:

生物技术是生物技术.
材料科学材料科学材料科学
纳米医学是一种纳米医学.

背景情况:

使者RNA (mRNA) 疗法需要高效和有针对性的输送系统.
阴离子聚合物显示出作为遗传载体的潜力,但在体内有效性和可变性较差.
需要一个通用功能化策略来提高阴离子聚合物输送能力.

研究的目的:

开发一种多功能策略,将阴离子聚合物转化为有效的mRNA输送载体.
为了提高mRNA输送的体内疗效和器官选择性,使用修饰的阴阳性聚合物.
证明这些改性聚合物在疫苗应用和与脂质纳米颗粒 (LNP) 结合中的潜力.

主要方法:

采用一种简单的功能化后方法,从商业化聚合物中制造脂和化聚合物 (PAP).
评估了PAP的增强细胞吸收,内体逃生和mRNA释放功能.
在体内对特定器官的输送进行了评估,包括单组件PAP的脏特异性输送和与PAP配制的四组件LNP的肺/肝特异性输送.
疫苗的有效性在小鼠黑色素瘤模型中得到验证.

主要成果:

PAPs在体内显著增强了mRNA表达,比未经修改的对应物高出30500倍.
一组件PAP实现了脏特异的mRNA输送.
与PAP配制的四组分LNP使肺和肝脏特异的mRNA输送成为可能.
这些器官选择性系统的性能优于现有的聚合物和LNP基准.

结论:

开发的策略提供了一种通用方法,可以从易于获得的阴离子聚合物中创建高效的mRNA载体.
脂和基聚合物 (PAP) 是一个有前途的平台,可以有效地提供器官选择性的mRNA.
这种方法对于需要器官向的mRNA疗法的临床转化具有相当大的潜力.

关键词:

阴离子聚合物的聚合物.脂质纳米颗粒的使用方法提供mRNA的交付.器官的选择性器官的选择性多功能修改策略的多功能修改策略.

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