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関連する概念動画

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

13.9K
To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
13.9K
RNA Editing02:23

RNA Editing

9.0K
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...
9.0K
CRISPR01:59

CRISPR

50.5K
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...
50.5K
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

6.0K
Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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関連する実験動画

Updated: Jun 24, 2025

Protein Transfection of Mouse Lung
04:21

Protein Transfection of Mouse Lung

Published on: May 15, 2013

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肺への遺伝子編集の流れ

Mattijs Bulcaen1, Marianne S Carlon1

  • 1Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium.

Science (New York, N.Y.)
|June 13, 2024
PubMed
まとめ

胞性線維症の遺伝子治療は 静脈内投与により 肺の自然な障壁を克服して 呼吸道基底細胞に到達できます このアプローチは この遺伝的肺疾患を 治療する新しい方法を提示しています

科学分野:

  • 肺医学
  • 遺伝子療法
  • 細胞生物学

背景:

  • 胞性線維症 (Cystic Fibrosis,CF) は,肺に影響する遺伝疾患で,粘液が濃くなり,
  • 現在の遺伝子療法の戦略は 治療用遺伝子を肺内の標的細胞に届けることに 課題があります
  • 肺の修復に不可欠な幹細胞であり,CF遺伝子治療の標的となる可能性があります.

研究 の 目的:

  • 呼吸道基底細胞を標的とした静脈内投与の有効性を調査する.
  • 胞性線維症の遺伝子治療における肺の障壁を克服するこの方法の能力を評価する.

主な方法:

  • 遺伝子治療薬の静脈内投与システムの開発
  • 胞性線維症の動物モデルを用いて投与システムをテストする.
  • 出産後の呼吸道ベース細胞における遺伝子発現と細胞標的を定量化する.

主要な成果:

  • 静脈内投与により,治療薬が呼吸道基底細胞に成功しました.
  • 呼吸道上皮質と粘液層を含む肺の障壁の重要な克服が観察されました.
  • 標的基底細胞における成功遺伝子発現の証拠が確認された.

さらに関連する動画

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

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CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
08:31

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation

Published on: June 20, 2019

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関連する実験動画

Last Updated: Jun 24, 2025

Protein Transfection of Mouse Lung
04:21

Protein Transfection of Mouse Lung

Published on: May 15, 2013

9.4K
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

33.9K
CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
08:31

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation

Published on: June 20, 2019

14.0K

結論:

  • 静脈内ターゲティングは,気管の基礎細胞に遺伝子治療を施すための有効な戦略です.
  • この方法は肺の遺伝子治療における 重要な障害を克服する見込みです
  • 胞性線維症の患者に対する効果的な治療法につながる可能性がある.