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Related Concept Videos

CRISPR01:59

CRISPR

50.9K
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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Updated: Jun 30, 2025

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes
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Efficient and multiplexed somatic genome editing with Cas12a mice.

Jess D Hebert, Haiqing Xu, Yuning J Tang

    Biorxiv : the Preprint Server for Biology
    |March 18, 2024
    PubMed
    Summary

    Researchers developed new mouse models for somatic genome editing using enhanced Acidaminococcus sp. Cas12a (enAsCas12a). This enables the creation of complex genetic changes in vivo, accelerating disease modeling and genetic interaction studies.

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    Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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    Area of Science:

    • Genetics and Genomics
    • Molecular Biology
    • Disease Modeling

    Background:

    • Somatic genome editing in mouse models advances understanding of in vivo genetic alterations.
    • Current models have limitations in creating multiple targeted edits, hindering the study of complex genetic interactions.
    • Understanding complex genetic interactions is crucial for development, homeostasis, and disease research.

    Purpose of the Study:

    • To accelerate and expand the generation of complex genotypes in somatic cells.
    • To develop novel transgenic mouse models for efficient somatic genome editing.
    • To facilitate high-throughput investigation of coincident genomic alterations in vivo.

    Main Methods:

    • Generated transgenic mice expressing enhanced Acidaminococcus sp. Cas12a (enAsCas12a) under Cre-regulated and constitutive control.
    • Utilized Cas12a with crRNA arrays containing tandem guides for multiplex gene targeting.
    • Integrated modular crRNA arrays with clonal barcoding to quantify tumor formation and crRNA efficiency.

    Main Results:

    • enAsCas12a-mediated somatic genome editing robustly generated compound genotypes in vivo.
    • Demonstrated the initiation of diverse cancer types driven by homozygous inactivation of tumor suppressor gene trios.
    • Successfully quantified tumor size, number, and crRNA efficiency using clonal barcoding.

    Conclusions:

    • The developed Cas12a mouse alleles enable rapid generation of complex genotypes in somatic cells.
    • These models significantly facilitate high-throughput investigation of coincident genomic alterations in vivo.
    • The study provides a powerful tool for advancing research in neuroscience, cancer biology, and beyond.