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Updated: Jan 7, 2026

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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在体内基因编辑用于脂质管理:针对ANGPTL3的CRISPR-Cas9治疗的早期观点

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
概括

No abstract available in PubMed .

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CRISPR

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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