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Genetically Engineered Mouse Models in Cancer Drug Development

Rutuja Sanjay Deshmukh


Higher rate of compounds entering clinical testing as effective anticancer drugs indicates necessity for better methods for preclinical studies. Therapeutic activity of compounds tested in xenograft mouse model and their efficacy in humans shows poor correlation, but it does not mean that genetically engineered mouse model will be of controlled use in drug development. Use of genetically engineered mouse model is one of the best solutions for human cancer. It facilitates the identification of the right target, right drug and the right patients. The use of tumor bearing genetically engineered mouse improves preclinical testing. These models summed up specific molecular pathways in tumor initiation and propagation. These also provides a biological system to study the disease process for generating new therapies and testing molecularly targeted drugs. In this review, we discuss the advantages and limitations of genetically engineered mice and better solutions for adapting these tumors for use in preclinical testing. In this article, we discuss uses of xenograft mouse models for cancer drug development, then describe the opportunities and challenges in the application of novel genetically engineered mouse models that mimics the genetic and biological evolution of human cancer. There are several applications in target validation, evaluation of tumor response, investigation of pharmacodynamics markers of drug action and understanding toxicity has the ability to markedly improve the success of cancer drug development. 


genetic engineering, mouse model, cancer, drug development

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