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生物响应化学聚合体中介持续和肝脏特异性siRNA转移在vivo中

Ri Huang¹, Feifei Wang², He Fu³

¹Biomedical Polymers Laboratory, College of Chemistry, Chemical Engineering and Materials Science, and State Key Laboratory of Radiation Medicine and Protection, Soochow University, Suzhou 215123, P. R. China.

Biomacromolecules

|October 23, 2023

概括

生物响应性基马基聚合体有效地传递小干扰RNA (siRNA) 进行肝脏特异性基因沉默 in vivo. 这种新的siRNA传递系统显示出治疗肝脏疾病的降低毒性的前景.

科学领域:

生物技术是生物技术.
纳米医学是一种纳米医学.
在RNA治疗方面,RNA疗法.

背景情况:

小干扰RNA (siRNA) 具有治疗潜力,但需要有效的,无毒的输送系统.
实现组织特异性输送,特别是到肝脏,仍然是siRNA疗法的挑战.

研究的目的:

开发和评估生物响应性基马基聚合体 (BCP) 以实现高效和肝脏特异性的siRNA输送.
为了评估BCP-siRNA在体外和体内用于ApoB mRNA沉默的疗效和安全性.

主要方法:

化学聚合体 (BCP) 与聚乙胺的内部外进行合成,用于siRNA封装.
描述了BCP-siRNA复合体的封装效率,粒子大小和电荷.
在HepG2细胞中进行了体外基因沉默,并在小鼠中进行了体内研究,以评估肝脏积累和ApoB mRNA下调.

主要成果:

BCP实现了高siRNA封装 (95-100%) 形成稳定,小 (55-64 nm),中性纳米粒子.
与没有细胞毒性的利波胺相比,BCP-siApoB在体外表现出更好的沉默 (93%在50nM) .
在体内研究表明,一次剂量后,肝脏的优先积累和持续的ApoBmRNA下调 (>90%长达10天) 持续下调.

结论:

生物反应性基马基聚合体是有效的,肝脏特异性的siRNA输送载体.
BCP-siRNA具有高疗效和低毒性,为基于siRNA的肝病治疗提供了一个有前途的平台.
进一步增强肝脏向和疗效被观察到与N-乙糖氨酸合.

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