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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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CRISPR01:59

CRISPR

58.1K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Homologous Recombination02:31

Homologous Recombination

64.1K
The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Updated: Feb 20, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
07:56

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

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CRISPR-Cas13によるRNA編集

David B T Cox1,2,3,4,5,6, Jonathan S Gootenberg1,2,3,4,7, Omar O Abudayyeh1,2,3,4,6

  • 1Broad Institute of Massachusetts Institute of Technology (MIT) and Harvard, Cambridge, MA 02142, USA.

Science (New York, N.Y.)
|October 27, 2017
PubMed
まとめ
この要約は機械生成です。

研究者はCRISPR-Cas13とADAR2を使用して,REPAIRと呼ばれる新しいRNA編集プラットフォームを開発しました. このシステムは 哺乳類の細胞のRNAレベルで 病気を引き起こす変異を 正確に修正し 治療的可能性を秘めています

さらに関連する動画

CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.

Published on: December 11, 2020

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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

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関連する実験動画

Last Updated: Feb 20, 2026

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
07:56

Genome Editing in Mammalian Cell Lines using CRISPR-Cas

Published on: April 11, 2019

23.3K
CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.
07:46

CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.

Published on: December 11, 2020

6.5K
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

35.9K

科学分野:

  • バイオテクノロジー
  • 分子生物学
  • 遺伝子工学

背景:

  • RNA編集は 病気に関連する配列を修正することで 遺伝疾患を治療するための有望な戦略を提供します
  • タイプVIのCRISPR-Casシステム,特にCas13酵素は,プログラム可能なRNA操作の可能性を持つRNA誘導核酸である.

研究 の 目的:

  • 堅固なRNAを 破壊するCas13の オーソログを設計する
  • RNA 2 (ADAR2) に作用するアデノシンデアミナーゼを誘導するために,触媒的に無効な Cas13 (dCas13) を使用して哺乳類の細胞におけるRNA編集を実証する.
  • 研究と治療のための多用途のRNA編集プラットフォームを開発する.

主な方法:

  • タイプVIのCRISPR-Casシステムのプロファイリング
  • 触媒的に不活性なCas13 (dCas13) 変種を設計する.
  • 標的型アデノシン- トゥー- イノシン (A- to- I) RNA編集のためにRNA2 (ADAR2) に作用するアデノシンデアミナーゼとdCas13の共発.
  • 高特異性の変種と 最小限のウイルス伝達システムの開発

主要な成果:

  • REPAIRシステムを使用して哺乳類の細胞でRNA編集が成功していることが実証されました.
  • 厳格な配列の制限なしに 病原性変異を持つ全長トランスクリプトを編集する能力を示した.
  • 高特異性の変種と ウイルスの伝達に適した コンパクトなシステムを設計した

結論:

  • REPAIRシステムは新しいプログラム可能なRNA編集プラットフォームです.
  • この技術は基礎研究,遺伝疾患の治療開発,バイオテクノロジーに広く適用できます.
  • REPAIRはDNAの修正を回避して RNAの配列を修正するための柔軟なアプローチを提供します.