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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...
6.4K
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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3.8K
MicroRNAs01:22

MicroRNAs

3.2K
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...
3.2K
Regulation of Expression Occurs at Multiple Steps02:24

Regulation of Expression Occurs at Multiple Steps

23.8K
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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Updated: Oct 15, 2025

Manipulation of Gene Function in Mexican Cavefish
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スタウディンガー還元によるMorpholino Antisense Oligonucleotide機能の小分子制御

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
PubMed
まとめ

研究者らは精密な遺伝子破壊のために,小分子トリガードケージモルフォリノアンチセンス剤 (cMO) を開発した. これらの新薬は 遺伝子発現の時間的・空間的な制御を可能にし 胚の発達の研究のための 進歩的なツールとなっています

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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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科学分野:

  • 分子生物学
  • 発達生物学
  • オリゴヌクレオチド化学

背景:

  • 条件付き活性化され,ケージ化されたモルフォリノアンチセンセス剤 (cMO) は,胚の発達中の遺伝子発現と機能を調査するために不可欠です.
  • 現在のcMOは通常,活性化のために光または酵素トリガーを必要とし,特定の生物学的文脈での適用を制限しています.

研究 の 目的:

  • 遺伝子ノックダウンのための最初の小分子反応性CMOを開発する.
  • ゼブラフィッシュの胚におけるフォスフィン誘発のCMOの有効性を実証する.

主な方法:

  • 化学活性化のための柔軟なリンク設計を用いた周期的なcMOの合成.
  • cMOsの迅速かつ効率的なデカージングのためのStaudingerの減少の適用.
  • cMOをゼブラフィッシュの胚で 2つの発達的に重要な遺伝子に対してテストする.

主要な成果:

  • 小分子反応性CMOの 合成に成功しました
  • ゼブラフィッシュの胚における標的遺伝子発現のフォスフィン誘発によるノックダウンが実証された.
  • 小分子を用いた生物対位遺伝子のノックダウンのための新しい方法を確立しました.

結論:

  • 小分子トリガーのcMOは 遺伝子ノックダウン技術の 重要な進歩を表しています
  • この新しいcMOのクラスは,発達研究における遺伝子発現の空間時間的な制御のためのツールキットを拡張します.
  • スタウディンガー還元ベースのデカージングは,アンチセンセスのエージェントの活性化のためのバイオオートゴーナルで効率的なアプローチを提供します.