Biomol Ther.  2014 Jul;22(4):267-274.

Genetically Engineered Mouse Models for Drug Development and Preclinical Trials

• Lee H

Abstract

Drug development and preclinical trials are challenging processes and more than 80% to 90% of drug candidates fail to gain approval from the United States Food and Drug Administration. Predictive and efficient tools are required to discover high quality targets and increase the probability of success in the process of new drug development. One such solution to the challenges faced in the development of new drugs and combination therapies is the use of low-cost and experimentally manageable in vivo animal models. Since the 1980's, scientists have been able to genetically modify the mouse genome by removing or replacing a specific gene, which has improved the identification and validation of target genes of interest. Now genetically engineered mouse models (GEMMs) are widely used and have proved to be a powerful tool in drug discovery processes. This review particularly covers recent fascinating technologies for drug discovery and preclinical trials, targeted transgenesis and RNAi mouse, including application and combination of inducible system. Improvements in technologies and the development of new GEMMs are expected to guide future applications of these models to drug discovery and preclinical trials.

Keyword

Genetically engineered mouse models; Preclinical trials; Drug discovery; Targeted transgenesis; RNAi mouse

MeSH Terms

Animals
Drug Discovery
Gene Transfer Techniques
Genome
Mice*
Models, Animal
United States Food and Drug Administration