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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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Related Experiment Video

Updated: Mar 21, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

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Engineering Delivery Vehicles for Genome Editing.

Christopher E Nelson1,2, Charles A Gersbach1,2,3

  • 1Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708.

Annual Review of Chemical and Biomolecular Engineering
|May 6, 2016
PubMed
Summary
This summary is machine-generated.

Genome engineering offers new gene therapy options, but safe delivery is key. Research is advancing precise genetic modifications for in vivo applications and therapeutic development.

Keywords:
CRISPRTALENsnanoparticlenonviral gene therapyviral gene therapyviruszinc finger nucleases

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Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Molecular Biology

Background:

  • Genome engineering technologies have advanced gene therapy possibilities, including disease models, cell therapies, and in vivo gene repair.
  • Clinical translation of genome engineering faces challenges in developing safe and effective delivery vehicles.
  • While in vitro applications are established, preclinical research is increasingly focused on in vivo genome editing.

Purpose of the Study:

  • To review the development of genome engineering platforms.
  • To discuss the prospects of viral and nonviral delivery vehicles for genome editing.
  • To highlight promising therapeutic applications of genome engineering.

Main Methods:

  • Summary of genome engineering platforms: meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas9.
  • Review of viral and nonviral delivery systems for genome editing tools.
  • Discussion of preclinical and clinical advancements in therapeutic applications.

Main Results:

  • Genome engineering platforms offer flexibility for precise genetic modifications.
  • Significant progress is being made in developing safe and effective delivery vehicles.
  • Promising preclinical data supports in vivo applications and therapeutic development.

Conclusions:

  • Genome engineering holds significant potential for gene therapy.
  • Overcoming delivery challenges is crucial for clinical translation.
  • Continued research in delivery systems and therapeutic applications will drive future advancements.