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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

772
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...
772
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...
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Related Experiment Video

Updated: Nov 2, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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Modulating Cas9 activity for precision gene editing.

Merve Uslu1, Pınar Siyah1, Andrew John Harvey2

  • 1Graduate School of Natural and Applied Sciences, Yeditepe University, Istanbul, Turkey; Department of Genetics and Bioengineering, Faculty of Engineering, Yeditepe University, Istanbul, Turkey.

Progress in Molecular Biology and Translational Science
|June 15, 2021
PubMed
Summary
This summary is machine-generated.

CRISPR/Cas9 gene editing shows promise but faces challenges with off-target effects. Pharmaceutical modulation of Cas9 activity is crucial for enhancing safety and enabling clinical applications in gene therapy.

Keywords:
Gene editingOff-target issuesspCas9

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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetics

Background:

  • CRISPR/Cas9 is a versatile RNA-guided nuclease for targeted DNA modification.
  • Off-target activity of Cas9 limits its therapeutic potential in genetic disease correction.
  • Current strategies focus on protein engineering and guide RNA selection to improve specificity.

Purpose of the Study:

  • To review recent advancements in precise gene editing technologies based on spCas9.
  • To discuss tools for modulating Cas9 activity and increasing CRISPR/Cas9 system fidelity.
  • To highlight the need for pharmaceutical interventions to enhance Cas9 safety for clinical use.

Main Methods:

  • Review of literature on spCas9 technologies and Cas9 modulating tools.
  • Analysis of strategies to improve specificity and efficiency of CRISPR/Cas9.
  • Discussion of potential pharmaceutical approaches for modulating Cas9 activity.

Main Results:

  • Recent developments in spCas9 offer enhanced precision in gene editing.
  • Various tools can modulate Cas9 activity to increase the fidelity of the CRISPR/Cas9 system.
  • Despite improvements, achieving clinical safety levels for Cas9 gene editing remains a challenge.

Conclusions:

  • Further research is needed to ensure the safety and efficacy of CRISPR/Cas9 for clinical applications.
  • Pharmaceutical modulation of Cas9 activity presents a promising avenue for robust and precise gene editing therapeutics.
  • Developing safe and effective gene editing strategies is essential for treating inherited diseases.