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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

535
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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CRISPR01:59

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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

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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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Homologous Recombination02:31

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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: Oct 5, 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

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Basic Principles and Clinical Applications of CRISPR-Based Genome Editing.

Jung Min Lim1, Hyongbum Henry Kim1,2,3,4,5,6,7

  • 1Department of Pharmacology, Yonsei University College of Medicine, Seoul, Korea.

Yonsei Medical Journal
|January 27, 2022
PubMed
Summary

CRISPR gene editing advances offer hope for genetic disorders. This review covers CRISPR-Cas9, base editors, and prime editors, highlighting their therapeutic potential despite ongoing safety concerns.

Keywords:
CRISPR-Associated Protein 9CRISPR-Cas systemsGenome editinggene editinggenetic therapy

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Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Sequencing technologies enable identification of genetic variants in diseases.
  • CRISPR-Cas9 technology has rapidly advanced since 2012.
  • Genetic disorders often lack effective treatment options.

Purpose of the Study:

  • To review the principles of CRISPR-based genome editing.
  • To explain mechanisms of novel editors like base and prime editors.
  • To evaluate therapeutic applications through clinical trials and animal studies.

Main Methods:

  • Literature review of CRISPR-Cas9 principles.
  • Explanation of base and prime editor mechanisms.
  • Analysis of recent clinical trials and animal studies on CRISPR therapies.

Main Results:

  • CRISPR technology has progressed significantly, offering hope for genetic diseases.
  • Base and prime editors represent advancements in genome editing precision.
  • Clinical trials and animal studies show promising therapeutic potential.

Conclusions:

  • CRISPR-based genome editing is a rapidly advancing field with significant therapeutic promise.
  • Efficacy and safety remain critical considerations for clinical application.
  • CRISPR technology is expected to become increasingly prevalent in clinical practice.