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Essential Knowledge Briefing: Genome Editing Applications for Disease Modeling and Cell Therapy

3D rendering of CRISPR-Cas9 and guide RNA interacting with DNA.

CRISPR-Cas technology is revolutionizing cell biology due to the ease and efficiency by which it enables the genetic manipulation of mammalian cells. Through targeted modification of specific genes or regulatory regions, researchers can now rapidly generate precise genetic models to study normal and diseased cell physiology. Beyond genetic manipulation for research purposes, CRISPR-Cas genome editing holds great potential for therapeutic applications.

This eBook serves to highlight the versatility and power of CRISPR-Cas genome editing when applied to cell culture systems for disease modeling and the development of cellular therapies.

The following areas are discussed:

  • Brief history of precise genome editing techniques, including CRISPR
  • Choosing the right CRISPR system for your experiment
  • Applications for CRISPR-Cas technology in culture systems
  • Current challenges and troubleshooting
  • Future directions of the field


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