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Related Concept Videos

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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CRISPR01:59

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CRISPR/Cas9 Genome Editing01:28

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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

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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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What is Genetic Engineering?00:49

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Related Experiment Video

Updated: Mar 18, 2026

Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells
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Using CRISPR/Cas9 Gene Editing to Investigate the Oncogenic Activity of Mutant Calreticulin in Cytokine Dependent Hematopoietic Cells

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CRISPR-Cas9 technology: applications and human disease modelling.

Raul Torres-Ruiz, Sandra Rodriguez-Perales

    Briefings in Functional Genomics
    |June 28, 2016
    PubMed
    Summary

    The CRISPR-Cas9 system enables precise genome engineering for diverse biomedical applications. This powerful gene editing tool facilitates disease modeling and advances our understanding of disease mechanisms and treatments.

    Keywords:
    CRISPRCas9animal modelsbiomedicinecancergene editinggenome engineeringhuman disease

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    Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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    Area of Science:

    • Biotechnology
    • Molecular Biology
    • Genomics

    Background:

    • Genome engineering is crucial for biomedical research and medicine.
    • The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 system has transformed gene editing.
    • CRISPR-Cas9 allows targeted double-strand breaks in various organisms and cell types.

    Purpose of the Study:

    • To highlight the revolutionary impact of CRISPR-Cas9 in genome engineering.
    • To showcase the diverse applications of CRISPR-Cas9 technology beyond basic gene editing.
    • To emphasize the potential of CRISPR-Cas9 in advancing disease modeling and understanding.

    Main Methods:

    • Utilizing CRISPR-Cas9 for targeted double-strand DNA breaks.
    • Applying CRISPR-Cas9 for gene expression regulation.
    • Employing CRISPR-Cas9 for epigenome editing and live-cell labeling.

    Main Results:

    • CRISPR-Cas9 facilitates efficient genome editing across diverse species and cell lines.
    • The technology enables gene expression regulation, epigenome modification, and chromosomal labeling.
    • CRISPR-Cas9 supports high-throughput screening and the creation of CRISPR-based disease models.

    Conclusions:

    • CRISPR-Cas9 technology offers versatile tools for studying gene function.
    • The system significantly enhances the generation of disease models for research.
    • Further research into CRISPR-Cas9 mechanisms and challenges will accelerate disease understanding and therapeutic development.