Drug discovery oncology in a mouse: concepts, models and limitations

Area of Science:

• Oncology
• Pharmacology
• Genetics

Background:

• Mouse models are essential in oncology drug discovery for target validation and preclinical assessments.
• Current murine models have significantly advanced understanding of drug mechanisms before clinical trials.
• Genome editing technologies, particularly CRISPR/Cas9, are revolutionizing preclinical model development.

Purpose of the Study:

• To review current mouse models used in oncology drug discovery.
• To highlight the impact and potential of CRISPR/Cas9 technology in creating novel preclinical models.

Main Methods:

• Review of existing literature on murine models in drug discovery.
• Discussion of advancements in genome editing, focusing on CRISPR/Cas9.
• Analysis of the application of CRISPR/Cas9 for *in vivo* preclinical model generation.

Main Results:

• Established mouse models have been instrumental in drug discovery workflows.
• CRISPR/Cas9 technology enables direct *in vivo* genome editing, expanding model capabilities.
• Novel CRISPR/Cas9-based models offer enhanced precision for preclinical research.

Conclusions:

• The strategic use of mouse models is fundamental to successful oncology drug discovery.
• CRISPR/Cas9 represents a significant advancement, providing powerful new tools for preclinical research.
• Future drug development will likely leverage these advanced genome-edited preclinical models.