用脂质替代聚乙烯胺基传递系统传递的治疗性小干扰RNA的生物分布

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Luis C Morales¹, Amarnath Rajendran¹, Aysha Ansari¹

¹Department of Chemical and Materials Engineering, Faculty of Engineering, University of Alberta, Edmonton, AB T6G 1H9, Canada.

Molecular pharmaceutics

|January 31, 2024

概括

脂质替代聚乙烯胺 (PEI) 载体在体内有效地将小干扰RNA (siRNA) 传递给白血病细胞和瘤. 这些新型Leu-Fect载体改善了siRNA生物分布,解决了siRNA疗法的关键挑战.

科学领域:

生物技术是生物技术.
纳米医学是一种纳米医学.
分子生物学分子生物学

背景情况:

小干扰RNAs (siRNAs) 是用于体外应用的强大的基因表达调节器.
显著的挑战阻碍了体内siRNA的治疗用途,包括降解,细胞吸收不足和有限的生物分布.

研究的目的:

评估三种基于脂质替代聚乙烯胺 (PEI) 的载体 (Leu-Fect A-C) 在体内siRNA传递效率.
在小鼠的静脉注射后,评估siRNA在各种器官和白血病异种移植瘤中的生物分布.

主要方法:

用 Leu-Fect 载体合成 siRNA 的纳米粒子.
在体内静脉注射给携带白血病的小鼠.
使用干环RT-PCR,qPCR和数字滴滴PCR进行siRNA量化.

主要成果:

Leu-Fect载体成功将siRNA输入K652和MV4-11白血病细胞和异种移植瘤中.
所有携带者都增强了siRNA传递到肝脏以外的器官,首选积累在肺和脏.
在测试的载体中,Leu-Fect A表现出最高的器官生物分布.

结论:

脂质替代PEI基载体在克服siRNA基疗法的药代动力学障碍方面表现有前途.
这些传递系统代表了siRNA疗法的临床转化潜在的进步.

关键词:

生物分销生物分销这是一种脂聚合物.纳米粒子是一种纳米粒子.药物动力学药物动力学 siRNA 是一个RNA.

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