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Cold Spring Harb Protoc. 2014 Feb 1;2014(2):131-43. doi: 10.1101/pdb.top069997.

Translational therapeutics in genetically engineered mouse models of cancer.

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Departments of Medicine and Pathology, Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center, New York, New York 10032;


Advances in knowledge of the molecular alterations of human cancers, refinements in technologies for the generation of genetically engineered mouse models (GEMMs), and the development of cancer therapies have accelerated in recent years. Progress in these fields provides the foundation for clinically relevant studies to be performed in GEMMs, through which it is possible to glean information on drug efficacy and to identify determinants of sensitivity and resistance to drugs and drug combinations. GEMMs used in pre-, co-, and postclinical studies must closely recapitulate the genetics, histopathology, and response to therapy of the human disease. Prevention and intervention trials can be designed in GEMMs to test the effects of drugs on tumor initiation, regression, and progression. Given their complexity, careful consideration of the infrastructure requirements and practical aspects of each individual experiment, including enrollment, tumor monitoring, and dose and schedule, must be considered in the design of therapeutic studies in GEMMs. Advantages of GEMMs include the ability to rapidly perform drug efficacy studies in a defined genetic background, the ease of pharmacodynamic and pharmacokinetic assessments, and the possibility of experimentally manipulating model systems to address questions that cannot be addressed in patients. In light of these features, GEMMs are useful tools for translational studies to inform clinical trials in cancer patients.

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