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相关概念视频

RNA Interference01:23

RNA Interference

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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...
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Experimental RNAi02:15

Experimental RNAi

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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...
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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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

Small interfering RNAs (siRNA)

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MicroRNAs01:22

MicroRNAs

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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...
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Regulation of Expression Occurs at Multiple Steps02:24

Regulation of Expression Occurs at Multiple Steps

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Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
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相关实验视频

Updated: Oct 15, 2025

Manipulation of Gene Function in Mexican Cavefish
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通过Staudinger还原来控制Morpholino反意义寡核酸功能的小分子

Kristie Darrah1, Joshua Wesalo1, Bradley Lukasak1

  • 1Department of Chemistry, University of Pittsburgh, Pittsburgh, Pennsylvania 15260, United States.

Journal of the American Chemical Society
|October 27, 2021
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概括

研究人员开发了小分子触发的子形态反意义剂 (cMO),用于精确的基因敲击. 这些新型药物能够控制基因表达的时间和空间,为胚胎发育研究提供了先进的工具.

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相关实验视频

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科学领域:

  • 分子生物学
  • 发育生物学
  • 氧核酸化学

背景情况:

  • 在胚胎发育过程中,有条件激活的,被关闭的形态反感剂 (cMO) 对于研究基因表达和功能至关重要.
  • 目前的cMO通常需要光或酶触发器来激活,这限制了它们在某些生物环境中的应用.

研究的目的:

  • 开发第一种用于基因破坏的小分子响应性cMO.
  • 在斑马鱼胚胎中证明素触发的cMO的有效性.

主要方法:

  • 使用灵活的链接设计进行化学激活的循环cMO的合成.
  • 应用Staudinger减肥法,以快速高效地减肥cMO.
  • 在斑马鱼胚胎中对两个与发育相关的基因进行cMO测试.

主要成果:

  • 成功合成了小分子响应的cMO.
  • 在斑马鱼胚胎中证明了素触发的目标基因表达.
  • 使用小分子建立了生物对角基因敲除的新方法.

结论:

  • 小分子触发的cMO代表了基因淘汰技术的重大进步.
  • 这种新的cMO类扩大了发育研究中基因表达的时空控制工具包.
  • 基于降解的Staudinger脱皮方法提供了一种生物对等和高效的方法来激活反感应剂.