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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

611
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...
611
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

17.8K
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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Related Experiment Video

Updated: Oct 12, 2025

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

Published on: May 25, 2018

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Modulating CRISPR/Cas9 genome-editing activity by small molecules.

Siwei Chen1, Deng Chen1, Bin Liu2

  • 1Department of Chemical and Pharmaceutical Biology, Groningen Research Institute of Pharmacy, University of Groningen, Groningen 9713 AV, the Netherlands.

Drug Discovery Today
|November 25, 2021
PubMed
Summary
This summary is machine-generated.

Small molecules can precisely control CRISPR/Cas9 gene editing by modulating Cas9 activity. This review explores clinical applications and mechanisms for enhancing precise gene modification efficiency and therapeutic potential.

Keywords:
Anti-CRISPRCRISPR/Cas9Genome editingSmall molecules

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Last Updated: Oct 12, 2025

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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetics

Background:

  • CRISPR/Cas9 gene editing is a key tool for DNA modification in research and medicine.
  • Current limitations include low precise gene modification efficiency and prolonged, uncontrolled Cas9 activity.
  • Overcoming these challenges is crucial for expanding CRISPR/Cas9's therapeutic applications.

Purpose of the Study:

  • To review small molecules that can precisely modulate CRISPR/Cas9 genome editing activity.
  • To discuss the mechanisms of action for these small molecules.
  • To identify potential therapeutic strategies for enhancing CRISPR/Cas9 precision.

Main Methods:

  • Literature review of small molecules targeting CRISPR/Cas9 activity.
  • Analysis of mechanisms of action for small-molecule modulators.
  • Evaluation of clinical potential for precise gene editing regulation.

Main Results:

  • Small molecules offer potential for precise control over CRISPR/Cas9 activity.
  • Direct-acting small molecules show promise for regulating Cas9 activity.
  • These modulators can enhance gene modification efficiency and control Cas9 duration.

Conclusions:

  • Small molecules are valuable tools for fine-tuning CRISPR/Cas9 genome editing.
  • Direct-acting small molecules are particularly suitable for precise Cas9 regulation.
  • Findings aid in discovering novel small-molecule enhancers and inhibitors for CRISPR/Cas9 systems.