SEMMs: Somatically Engineered Mouse Models. A New Tool for In Vivo Disease Modeling for Basic and Translational...

Area of Science:

• Oncology
• Genetics
• Translational Medicine

Background:

• Most experimental cancer therapies fail in clinical trials due to inadequate preclinical models.
• Current xenograft models lack patient heterogeneity and immune systems, limiting translatability.
• Genetically Engineered Mouse Models (GEMMs) are costly and complex, hindering broad application.

Purpose of the Study:

• To review the limitations of current preclinical cancer models.
• To introduce Somatically Engineered Mouse Models (SEMMs) as an advancement.
• To highlight SEMMs as a platform for in vivo functional genomics (DIVoS).

Main Methods:

• Utilizing CRISPR/Cas9 genome editing technology.
• Introducing specific oncogenic signatures directly into somatic cells of target organs in post-natal animals.
• Bypassing the need for costly conventional genetically engineered mouse model strains.

Main Results:

• SEMMs provide a more accurate recapitulation of human disease heterogeneity and response.
• SEMMs enable direct in vivo functional genomics studies (DIVoS).
• SEMMs offer a cost-effective and efficient alternative to traditional GEMMs.

Conclusions:

• SEMMs represent a novel and powerful category of preclinical cancer models.
• SEMMs enhance the translatability of preclinical findings to clinical settings.
• SEMMs facilitate efficient in vivo drug screening and functional genomics research.