相关实验视频
Updated: Jul 28, 2025
08:31
Porous Silicon Microparticles for Delivery of siRNA Therapeutics
Published on: January 15, 2015
11.0K
一个模块化的RNA输送系统,包括球形核酸,构建在能逃脱内体的聚合物纳米粒子上
Antonio Garcia-Guerra1,2,3,4, Ruth Ellerington2,4, Jens Gaitzsch5,6
1Department of Physics, Clarendon Laboratory, University of Oxford Parks Road Oxford OX1 3PU UK.
Nanoscale advances
|June 1, 2023
概括
研究人员开发了新型聚合体球形核酸 (SNA) 来增强核酸输送. 这些pH敏感的聚合体有效地逃脱了内分泌体,克服了针对性基因疗法对像ALS这样的疾病的关键挑战.
科学领域:
- 生物材料科学 生物材料科学
- 纳米技术 纳米技术
- 基因治疗 基因治疗
背景情况:
- 核酸治疗需要有效的输送系统来克服生物障碍.
- 传统的球状核酸 (SNAs) 面临着内体捕获和降解的挑战.
- 针对特定的细胞和避免免疫系统清除对于治疗疗效至关重要.
研究的目的:
- 开发一种替代性的SNA核心,以改善内体体逃生和治疗性输送.
- 为了研究pH敏感的双块共聚物PMPC25-PDPA72用于自组装的核酸载体.
- 评估这些聚合SNAs在运动神经元中传递siRNA对抗C9orf72的疗效.
主要方法:
- PMPC25-PDPA72双块共聚合物的自组装成pH敏感的聚合物体.
- DNA寡核酸的结合,形成具有外部和内部冠状的聚合SNA.
- 针对特定细胞输送的和siRNA载荷的附着.
- 在体外输送向C9orf72的siRNA到类似运动神经元的细胞.
主要成果:
- 聚合物SNAs形成了带有DNA冠状的囊泡 (聚合体),能够携带核酸货物.
- 这些聚合体体通过pH触发的分解证明了内在的内分体逃脱.
- 针对C9orf72向运动神经元的siRNA的向输送,在低颗粒剂量下实现了有效的基因敲击.
结论:
- 敏感于pH的聚合物SNA为基于纳米粒子的SNA提供了一个有希望的替代方案,用于核酸输送.
- 开发出来的聚合体克服了内体捕获,增强了治疗潜力.
- 这个平台显示了针对性基因治疗的重大前景,以C9orf72siRNA用于ALS的传递为例.
更多相关视频
相关概念视频
02:15Experimental RNAi
6.2K
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...
6.2K
01:23RNA Interference
26.1K
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
26.1K
02:22Regulated mRNA Transport
6.3K
In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing...
6.3K
02:31Nuclear Export of mRNA
7.7K
Before mRNAs are exported to the cytoplasm, it is crucial to check each mRNA for structural and functional integrity. Eukaryotic cells use several different mechanisms, collectively known as mRNA surveillance, to look for irregularities in mRNAs. Irregular or aberrant mRNA are rapidly degraded by various enzymes. If a defective mRNA escapes the surveillance, it would be translated into a protein which would either be non-functional or not function properly. One of the primary irregularities in...
7.7K