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相关概念视频

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

27
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...
27
CRISPR01:59

CRISPR

52.1K
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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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...
17.0K
Homologous Recombination02:31

Homologous Recombination

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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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相关实验视频

Updated: Jul 14, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

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基因组和转录基因组工程通过紧和多功能CRISPR-Cas系统.

Guillermo Aquino-Jarquin1

  • 1RNA Biology and Genome Editing Section. Research on Genomics, Genetics, and Bioinformatics Laboratory. Hemato-Oncology Building, 4th Floor, Section 2. Children's Hospital of Mexico, Federico Gómez, Mexico City, Mexico.

Drug discovery today
|October 5, 2023
PubMed
概括

微小的CRISPR-Cas系统,包括微型的Cas9,Cas12和Cas13蛋白质,提供高效的基因组编辑. 它们的紧尺寸非常适合开发基于CRISPR的疗法,以克服传递挑战.

科学领域:

  • 分子生物学分子生物学
  • 遗传学 是一个遗传学.
  • 生物技术是生物技术.

背景情况:

  • 细菌免疫系统的集群定期间隔的短Palindromic重复 (CRISPR) 为真核生物基因组操纵提供了效应器.
  • 最近发现了较小的Cas蛋白 (迷你Cas9,Cas12,Cas13) 已被验证为人类细胞中的基因组和基因编辑.

研究的目的:

  • 突出用于治疗应用的紧CRISPR-Cas效应器的潜力.
  • 强调它们在克服基因组编辑的体内传递约束方面的优势.

主要方法:

  • 进行比较基因组学以识别新型CRISPR效应器.
  • 验证微型Cas蛋白 (Cas9,Cas12,Cas13) 作为人类细胞中的基因组和基因编辑工具.

主要成果:

  • 微型CRISPR-Cas蛋白在人类细胞中显示出高效的基因组编辑和基因编辑能力.
  • 这些系统的紧性质通过缓解交付挑战,促进了潜在的治疗策略.
  • 这些工具可以在不诱导染色体插入或基因组改变的情况下进行RNA敲除.

结论:

  • 微小的CRISPR-Cas系统代表了CRISPR工具包的显著扩展.
关键词:
在CRISPR-Cas系统中.编辑RNA的RNA编辑紧的核酶可以形成紧的核酶.基因治疗的基因疗法基因组编辑 基因组编辑

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  • 它们的可编程性,特异性和效率为新的治疗机会提供了有希望的平台.
  • 这些系统在临床环境中具有编辑致病突变和调节RNA的潜力.