相关实验视频

Updated: May 25, 2025

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

基于可电离性脂质纳米颗粒和阴离子聚合物载体的siRNA的向传递系统

Ziying Yao¹, Taiqing Liu¹, Jingwen Wang¹

¹Department of Pharmacy, Cancer Center and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China.

Biotechnology advances

|February 27, 2025

概括

小干扰RNA (siRNA) 疗法在治疗疾病方面显示出很大的前景. 本次审查突出了针对性传递系统的进展,重点关注可电离性脂质纳米颗粒 (LNP) 和性聚合物 (CP).

科学领域:

生物技术和生物医学工程生物技术和生物医学工程
分子生物学和遗传学分子生物学和遗传学
药物输送系统药物输送系统

背景情况:

小干扰RNA (siRNA) 为治疗应用提供序列特定的基因沉默.
到2024年批准的六种基于siRNA的药物显示出显著的治疗潜力.
siRNA疗法正在迅速发展,特别是有针对性的输送系统.

研究的目的:

审查针对性siRNA传递系统的最新进展.
专注于电离性脂质纳米粒子 (LNPs) 和阴离子聚合物 (CP) 载体.
讨论有针对性的修改策略及其在增强siRNA可药性方面的作用.

主要方法:

对针对性siRNA传递的最新文献的审查.
专注于使用可电离性脂质纳米颗粒 (LNP) 和性聚合物 (CP) 的输送系统.
基于目标修改的传递系统的分类 (配体,,抗体,体,氨基酸).

主要成果:

针对性策略的进展显著提高了siRNA的功效,降低了毒性.
可电离的LNP和CP是有针对性的siRNA传递的关键载体.
不同的向分量提高了细胞外和细胞内传递效率.

结论:

针对性交付对于克服siRNA相关挑战和提高治疗疗效至关重要.
可电离的LNP和CP代表了下一代siRNA治疗的有希望的平台.
对挑战和机遇的进一步研究将推动siRNA传递系统的发展.

关键词:

阴离子聚合物阴离子聚合物临床试验中的临床试验.可电离的脂质纳米颗粒消息传递者RNA是什么意思这是一种RNA干扰.小的干扰RNA可以干扰RNA.有针对性的交付目标.

更多相关视频

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

相关概念视频

siRNA - Small Interfering RNAs

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...

Experimental RNAi

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...