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Updated: May 6, 2026
A Protocol for Using Gene Set Enrichment Analysis to Identify the Appropriate Animal Model for Translational Research
Published on: August 16, 2017
Effective utilization and appropriate selection of genetically engineered mouse models for translational integration
Cory Abate-Shen1, Pier Paolo Pandolfi
1Departments of Urology and of Pathology and Cell Biology, Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center, New York, New York 10032;
Abstract:
The landscape of cancer research and therapy has radically changed over the past decades in at least two major respects: Our ability to model cancer in the mouse has risen to an unprecedented level of accuracy at the same time that novel cancer drugs have been developed in record numbers. This has led to an explosion in genetically engineered mouse model (GEMM) research, as GEMMs can potentially be used to test and optimize drugs in a variety of ways: preclinically (before testing in human patients), coclinically (in parallel with human testing), and postclinically (to optimize standard of care therapy). Thus the potential applications of faithful GEMMs of cancer have expanded from analysis of causal relationships between genetic aberrancies and tumorigenesis in preclinical efforts to a more comprehensive and systematic utilization of GEMMs for drug testing and clinical trial optimization. As GEMM research has grown, however, few standard protocols have been put in place regarding GEMM trials done in parallel with human trials (the "coclinical" approach), or in situations in which the available cohort of human patients is too small for valid statistical analysis. The success of such efforts will require an increased attention to the rigor with which mouse and human clinical efforts are designed, executed, and integrated.
Insights
Genetically engineered mouse models (GEMMs) revolutionize cancer research and drug development. Standardized protocols are needed for rigorous coclinical and small-cohort studies to optimize cancer therapy.
Area of Science:
- Oncology
- Translational Cancer Research
- Preclinical Cancer Models
Background:
- Cancer research has advanced significantly with improved mouse models and novel drug development.
- Genetically Engineered Mouse Models (GEMMs) offer high accuracy for cancer modeling.
- GEMMs are increasingly used for preclinical, coclinical, and postclinical drug testing.
Purpose of the Study:
- To highlight the expanded applications of GEMMs in cancer research and drug optimization.
- To address the lack of standardized protocols for coclinical GEMM trials and small patient cohorts.
- To emphasize the need for rigorous integration of mouse and human clinical studies.
Main Methods:
- Review of advancements in cancer modeling and drug development.
- Analysis of the utilization of GEMMs in preclinical, coclinical, and postclinical settings.
- Identification of challenges in current GEMM research protocols, particularly for coclinical trials.
Main Results:
- GEMMs have evolved from studying tumorigenesis to comprehensive drug testing and clinical trial optimization.
- There is a notable absence of standardized protocols for coclinical GEMM trials and small-scale human studies.
- The integration of mouse and human clinical efforts requires enhanced rigor in design and execution.
Conclusions:
- Faithful GEMMs are crucial for advancing cancer therapy and drug development.
- Standardized protocols are essential for the successful implementation of coclinical studies and small-cohort analyses.
- Increased rigor in designing and executing integrated mouse and human studies is vital for optimizing cancer treatment.
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