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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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...
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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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The Antiviral System of Bacteria and Archaea: CRISPR01:23

The Antiviral System of Bacteria and Archaea: CRISPR

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CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats is a adaptive immune system found in bacteria and archaea that protects against viral infections. This system enables prokaryotic cells to identify, remember, and neutralize foreign genetic elements, primarily bacteriophages, by storing fragments of the invader’s DNA as a genetic memory.The CRISPR immune response begins during an initial infection. Cas (CRISPR-associated) proteins play a central role in this...
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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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Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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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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Related Experiment Video

Updated: Jul 30, 2025

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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CRISPR-Cas System: The Current and Emerging Translational Landscape.

Narendranath Bhokisham1, Ethan Laudermilch1, Lindsay L Traeger1

  • 1Corporate Research Material Labs, 3M Center, 3M Company, Maplewood, MN 55144, USA.

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|May 16, 2023
PubMed
Summary

CRISPR-Cas technology revolutionizes medicine by enabling precise DNA editing for treating genetic diseases like sickle cell anemia and complex conditions such as cancer. This review explores current clinical trials, emerging CRISPR tools, and their use in disease modeling.

Keywords:
CRISPR animal modelsCRISPR-Casbase editorsclinical studygene editingprime editorstranslational research

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Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Translational Medicine

Background:

  • CRISPR-Cas technology offers unprecedented capabilities for editing human DNA.
  • This has profound implications for treating both congenital and acquired human diseases.

Purpose of the Study:

  • To review the current clinical trial landscape for CRISPR-Cas therapeutics.
  • To explore novel CRISPR-Cas-based tools and their therapeutic potential.
  • To discuss the application of CRISPR-Cas in disease modeling for preclinical research.

Main Methods:

  • Review of current clinical trials utilizing CRISPR-Cas systems.
  • Exploration of emerging CRISPR-based technologies (base editing, prime editing, transcriptional/epigenome/RNA editing).
  • Discussion of CRISPR-Cas applications in generating large animal disease models.

Main Results:

  • CRISPR-Cas therapies are advancing towards clinical application for monogenic and complex diseases.
  • New CRISPR tools expand the scope and precision of gene editing and regulation.
  • CRISPR-Cas facilitates the creation of relevant animal models for therapeutic development.

Conclusions:

  • CRISPR-Cas technology is a transformative force in medicine, with ongoing clinical trials demonstrating therapeutic promise.
  • Advancements in CRISPR tools are broadening therapeutic possibilities and applications.
  • The integration of CRISPR-Cas in disease modeling accelerates preclinical research and therapeutic innovation.