関連する実験動画

Updated: Jan 7, 2026

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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脂質管理のためのインビボ遺伝子編集: ANGPTL3を標的としたCRISPR-Cas9療法からの初期の展望

Luke J Laffin1, Steven E Nissen1

  • 1Section of Preventive Cardiology and Rehabilitation, Department of Cardiovascular Medicine, Cleveland Clinic Foundation, Cleveland, OH, USA; Cleveland Clinic Coordinating Center for Clinical Research, Cleveland Clinic Foundation, Cleveland, OH, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|January 4, 2026
PubMed
まとめ

No abstract available in PubMed .

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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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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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