RNA Interference as a Tool for Producing Knockdown Mice

Analysis at the individual level using genetically-engineered mice has allowed conclusions to be reached regarding the actual function of a target gene. In recent years, the “knockdown” method using RNA interference (RNAi) has been established as a powerful tool for analyzing gene function. In this review, we focus on RNAi knockdown technology for producing genetically-engineered mice and describe the value of this approach from the perspective of both basic research and therapeutic potential. First, we introduce the basic mechanism of RNAi and development of knockdown animals from worms to mice. Next, we describe strategies to produce knockdown mice using DNA-based expression vectors introduced into zygotes or embryonic stem cells. Finally, we refer to the trends of research for clinical application. By way of illustration, we show the production of knockdown mice for treatment of neurodegenerative disease and mention the prospect of therapeutic potential of RNAi technology.