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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

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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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Related Experiment Video

Updated: Jul 14, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
08:20

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization

Published on: September 2, 2021

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Genome and transcriptome engineering by compact and versatile CRISPR-Cas systems.

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
Summary

Tiny CRISPR-Cas systems, including miniature Cas9, Cas12, and Cas13 proteins, offer efficient genome editing. Their compact size is ideal for developing CRISPR-based therapeutics to overcome delivery challenges.

Keywords:
CRISPR-Cas systemsRNA editingcompact nucleasesgene therapygenome editing

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Related Experiment Videos

Last Updated: Jul 14, 2025

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) bacterial immune systems provide effectors for eukaryotic genome manipulation.
  • Discovery of smaller Cas proteins (miniature Cas9, Cas12, Cas13) has been recently validated for genome and base editing in human cells.

Purpose of the Study:

  • To highlight the potential of compact CRISPR-Cas effectors for therapeutic applications.
  • To emphasize their advantages in overcoming in vivo delivery constraints for genome editing.

Main Methods:

  • Comparative genomics to identify novel CRISPR effectors.
  • Validation of miniature Cas proteins (Cas9, Cas12, Cas13) as genome and base editing tools in human cells.

Main Results:

  • Miniature CRISPR-Cas proteins demonstrate efficient genome editing and base editing capabilities in human cells.
  • The compact nature of these systems facilitates potential therapeutic strategies by easing delivery challenges.
  • These tools enable RNA knockdown without inducing chromosomal insertions or genome alterations.

Conclusions:

  • Tiny CRISPR-Cas systems represent a significant expansion of the CRISPR toolkit.
  • Their programmability, specificity, and efficiency offer promising platforms for novel therapeutic opportunities.
  • These systems hold potential for editing pathogenic mutations and modulating RNA in clinical settings.