Gene silencing through RNA interference: Potential for therapeutics and functional genomics

A major focus of biology is determining the role that specific genes play within a cell. The ability to control gene expression is a powerful tool for biologists, and methods that facilitate the expression of genes in trans have been very useful in delineating gene function. However, methods that enable researchers to decrease or inhibit gene expression in a controlled and specific manner have been lacking. A significant advancement in the field of gene silencing is the recent discovery of RNA interference (RNAi), a post-transcriptional gene silencing mechanism first described in C. elegans and Drosophila and more recently shown to occur in mammals, including mouse and human cells. This chapter will examine several aspects of RNAi and its related gene silencing, mechanisms and will discuss the impact that technologies based on these endogenous cellular processes have had on studies of gene function and on new approaches to the treatment of disease.