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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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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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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Related Experiment Video

Updated: Apr 25, 2026

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
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Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.

Ami M Kabadi1, David G Ousterout1, Isaac B Hilton1

  • 1Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.

Nucleic Acids Research
|August 15, 2014
PubMed
Summary
This summary is machine-generated.

Researchers developed a lentiviral system for multiplex CRISPR/Cas9 genome engineering. This tool enables simultaneous gene editing and transcriptional activation for advanced biomedical research.

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Area of Science:

  • Molecular Biology
  • Gene Editing Technologies

Background:

  • CRISPR/Cas9 systems enable genome and transcriptome manipulation for biomedical research.
  • Multiplex genome engineering using CRISPR/Cas9 allows simultaneous targeting of multiple genomic sites.

Purpose of the Study:

  • To develop a uniform and sustained delivery system for multiplex CRISPR/Cas9 tools.
  • To enable efficient multiplex genome engineering and transcriptional regulation in human cells.

Main Methods:

  • A single lentiviral vector system was engineered to express a Cas9 variant, reporter gene, and up to four single guide RNAs (sgRNAs).
  • Golden Gate cloning was utilized for convenient incorporation of sgRNAs into the lentiviral vector.
  • The system was tested for its ability to mediate multiplex gene editing and transcriptional activation in immortalized and primary human cells.

Main Results:

  • The lentiviral system efficiently expressed Cas9 variant and multiple sgRNAs.
  • Multiplex gene editing and sustained transcriptional activation were successfully demonstrated in various human cell types.
  • The system provides uniform and sustained delivery of multiplex CRISPR/Cas9 tools.

Conclusions:

  • The developed lentiviral delivery system effectively supports multiplex CRISPR/Cas9 genome engineering.
  • This technology has the potential to advance CRISPR/Cas9 applications across diverse cell types.
  • The system facilitates simultaneous gene editing and transcriptional activation for enhanced research capabilities.