单个siRNA纳米囊用于增强RNAi传递
Ming Yan1, Min Liang, Jing Wen
1Department of Microbiology, Immunology, and Molecular Genetics, and California NanoSystems Institute (CNSI), University of California, Los Angeles, California 90095, USA.
Journal of the American Chemical Society
|August 8, 2012
概括
这项研究引入了一种新型纳米囊,用于输送单个小干扰RNA (siRNA) 分子,增强稳定性和细胞吸收,用于治疗疾病的潜在治疗应用.
科学领域:
- 生物技术是生物技术.
- 纳米技术 纳米技术
- 分子生物学分子生物学
背景情况:
- 合成小干扰RNA (siRNA) 显示出治疗前景,但在稳定性和传递方面面临挑战.
- 有效地将RNA干扰 (RNAi) 化合物输送到向组织和细胞仍然是临床转化的一个重大障碍.
研究的目的:
- 为单个siRNA分子开发和验证一种基于纳米囊的新型输送系统.
- 为了证明该系统在降低特定基因标的有效性,使用CCR5作为HIV治疗的模型.
主要方法:
- 在可降解的聚合物纳米囊中封装单个siRNA分子 (约. 20纳米直径,正表面电荷).
- 将CCR5-siRNA纳米囊输入293T细胞,以评估CCR5RNA下调.
- 在存在RNase和人血清时评估纳米囊稳定性和siRNA完整性.
- 基因沉默效率与自由siRNA和利波胺介导输送的比较.
主要成果:
- 纳米囊成功输送了CCR5-siRNA,在293T细胞中降低了CCR5RNA表达的调节至8%.
- 纳米囊保护siRNA免受RNase和血清的降解,保持完整性1小时.
- 在血清中,纳米囊输送的siRNA达到了<15%的CCR5-mCherry表达,显著优于利波胺 (55%).
结论:
- 一个新的单个siRNA纳米囊输送平台已经开发出来.
- 这项技术提供了增强的稳定性和有效的基因沉默,解决了当前siRNA疗法的关键局限性.
- 纳米囊平台在各种人类疾病中具有治疗开发的潜力.
相关概念视频
01:23RNA Interference
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...
01:23RNA Interference
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...
02:30siRNA - 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 ATP-dependent...
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 ATP-dependent...
02:15Experimental 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...
02:30Small interfering RNAs (siRNA)
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 ATP-dependent...
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 ATP-dependent...