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相关概念视频

MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
RNA Interference01:23

RNA 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...
RNA Interference01:23

RNA 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...
siRNA - Small Interfering RNAs02:30

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 ATP-dependent...
Experimental RNAi02:15

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...
Small interfering RNAs (siRNA)02:30

Small 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...

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相关实验视频

Updated: Jul 17, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

小RNA:RNA干扰可以用于治疗吗?

Nathan R Wall1, Yang Shi

  • 1Department of Pathology, Harvard Medical School, Boston, MA 02115, USA.

Lancet (London, England)
|October 31, 2003
PubMed
概括

RNA干扰 (RNAi) 提供了一种强大的基因沉默方法,小干扰RNA (siRNAs) 显示出基因治疗的前景. 对于临床应用,需要进一步开发交付和稳定性.

科学领域:

  • 分子生物学分子生物学
  • 遗传学 是一个遗传学.
  • 生物技术是生物技术.

背景情况:

  • RNA干扰 (RNAi) 是一种基因沉默的自然过程.
  • 小干扰RNAs (siRNAs) 是RNAi的关键作用因子,使序列特定的基因被淘汰.
  • 正在探索RNAi在病毒感染,癌症和遗传疾病中的治疗应用.

研究的目的:

  • 探索RNA干扰 (RNAi) 对于基因功能研究和治疗应用的潜力.
  • 为了研究小干扰RNAs (siRNAs) 用于向基因沉默的使用.
  • 通过研究参与癌症相关途径的基因家族来确定新的治疗点.

主要方法:

  • 利用合成小干扰RNAs (siRNAs) 进行序列特异性基因抑制.
  • 采用等离子体和病毒载体来有效地表达siRNA和短RNA (shRNA).
  • 开发RNA干扰载体以抑制特定的基因家族,如去无化酶.

主要成果:

  • 证明siRNAs可以选择性地抑制内源基因表达.
  • 确定了圆柱形瘤瘤抑制基因 (CYLD) 作为NF-kappaB激活和亡抵抗的调节者.
  • 在体外和体内使用基于等离子体和病毒载体的shRNA表达系统展示了稳定的基因表达抑制.

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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells
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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

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Last Updated: Jul 17, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

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Practical Use of RNA Interference: Oral Delivery of Double-stranded RNA in Liposome Carriers for Cockroaches
08:26

Practical Use of RNA Interference: Oral Delivery of Double-stranded RNA in Liposome Carriers for Cockroaches

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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells
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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

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结论:

  • RNAi,特别是使用siRNA和shRNA,为基因功能分析和潜在的治疗干预提供了强大的工具.
  • 有效和稳定的siRNA/shRNA向点组织的传递仍然是临床转化的一个关键领域.
  • 进一步的研究和传递系统的改进正在为利用基于RNAi的疗法进行临床试验铺平道路.