From Gene Targeting to Genome Editing: Transgenic animals applications and beyond

Area of Science:

• Molecular Biology
• Genetics
• Biomedical Research

Background:

• Genome modification technologies have evolved significantly over three decades.
• Advances allow systematic interrogation of gene function and modeling of genome's influence on cellular physiology.
• Homologous recombination (HR) was the standard for genomic modification.

Purpose of the Study:

• Provide a historical perspective on genome modification methods.
• Focus on the development and application of transgenic mice models.
• Discuss new nuclease-guided genome editing techniques and their impact.

Main Methods:

• Historical review of genome modification techniques.
• Focus on transgenic mice models.
• Description of nuclease-guided methods: Zinc Finger Nucleases (ZFN), Transcription Activator-Like Effector Nucleases (TALEN), and CRISPR/Cas systems.

Main Results:

• Significant progress in animal transgenesis and genome editing.
• Emergence of ZFN, TALEN, and CRISPR/Cas technologies.
• New perspectives opened for biomedical research and disease modeling.

Conclusions:

• Nuclease-guided genome editing represents a paradigm shift in molecular biology.
• These technologies offer powerful applications for biomedical research.
• Future directions involve refining techniques and expanding applications.