基托桑及其结构修饰用于siRNA传递
Mona Y Al-Absi1, Anna Eleonora Caprifico1, Gianpiero Calabrese1
1School of Life Sciences, Pharmacy and Chemistry, Kingston University London, Penrhyn Road, Kingston upon Thames, KT1 2EE, United Kingdom.
Advanced pharmaceutical bulletin
|June 21, 2023
概括
修改后的奇托在癌症基因治疗中显示出作为小干扰RNA (siRNA) 的有效载体的前景. 化学修饰增强了奇托桑的作用.
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:
- 使用小干扰RNA (siRNA) 的RNA干扰 (RNAi) 是癌症基因疗法的有效策略.
- 将完整的siRNA有效地输入目标细胞对于成功的基因沉默至关重要.
- 可生物降解的聚合物奇托被探索为siRNA传递的非病毒载体,但面临着像低转染效率和可溶性等局限性.
研究的目的:
- 审查最近对奇托的化学修改,以改善其作为siRNA传递载体的特性.
- 分析这些修改对物理化学性质,siRNA结合和纳米粒子特征的影响.
- 评估基托基改性siRNA输送系统的体外和体内性能.
主要方法:
- 关于用于siRNA传递的奇托最近化学修饰的文献综述.
- 讨论改性基托的化学结构和由此产生的物理化学性质.
- 在复杂化,细胞吸收,稳定性,细胞毒性和转染效率方面,基托基改性纳米颗粒与未经改性基托的比较.
主要成果:
- 已经研究了各种化学修饰,以克服奇托桑在siRNA传递方面的局限性.
- 修改后的奇托会表现出改变的物理化学特性,影响siRNA结合和纳米粒子形成.
- 性能评估显示,对某些改性基托衍生物的细胞吸收,血清稳定性和基因转染效率有显著改善.
结论:
- 素的化学修饰对于开发癌症基因治疗的有效siRNA输送系统至关重要.
- 特定的修改提供了增强的特性,从而提高了基因沉默功效.
- 需要进一步进行批判性分析,以确定未来临床应用中最有前途的奇多衍生物.
相关概念视频
02:30siRNA - Small Interfering RNAs
16.9K
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...
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...
16.9K
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
02:30Small interfering RNAs (siRNA)
3.6K
3.6K