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相关概念视频

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.
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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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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...
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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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相关实验视频

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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基因编辑流向肺部

Mattijs Bulcaen1, Marianne S Carlon1

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

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

基因治疗囊性纤维化现在可以通过静脉输入到达呼吸道基底细胞, 成功克服自然的肺障碍. 这种方法为治疗这种遗传性肺病提供了一种新途径.

科学领域:

  • 肺部医学
  • 基因治疗
  • 细胞生物学

背景情况:

  • 囊性纤维化 (CF) 是一种影响肺部的遗传疾病,导致粘液积聚.
  • 目前的基因疗法在将治疗基因传递到肺内向细胞方面面临挑战.
  • 呼吸道基底细胞是肺修复的关键干细胞,也是CF基因治疗的潜在目标.

研究的目的:

  • 调查静脉输送对呼吸道基底细胞的有效性.
  • 评估这种方法在囊性纤维化中克服肺部障碍的能力.

主要方法:

  • 开发一种静脉注射基因治疗药物的系统.
  • 使用囊性纤维化动物模型来测试输送系统.
  • 在分娩后的呼吸道基底细胞中量化基因表达和细胞向.

主要成果:

  • 静脉注射成功地将治疗药物输送到呼吸道基底细胞中.
  • 观察到肺部障碍物,包括呼吸道上皮和粘液层的显著克服.
  • 在向基底细胞中成功表达的证据得到证实.

结论:

  • 静脉向是一种可行的基因治疗策略,可以在囊性纤维化患者的呼吸道基底细胞中使用.

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  • 这种方法有望克服肺部基因疗法的关键障碍.
  • 进一步开发可能会为囊性纤维化患者提供有效的治疗方法.