Loss-of-function approaches such as gene knock-out or gene silencing are extremely powerful strategies for assigning function to a gene and for mapping the interconnections of intracellular regulatory pathways. Post-transcriptional gene silencing can be obtained via activation of a double-stranded RNA (dsRNA) mediated mechanism termed RNA interference (RNAi). RNAi has revealed an extremely versatile tool in Biomedical research that can be used in both single silencing gene experiments and in large-scale Functional Genomics studies and has been used as a tool for gene therapy. In the present paper the authors discuss the intracellular mechanisms underlying the RNAi phenomenon, as well the different strategies and their limitations for RNAi gene silencing in mammalian cells. The use of RNAi in the treatment of human diseases and in the investigation of both physiology and pathophysiology of adrenal cortex has also been reviewed.
Gene silencing; RNA interference; Functional genomics; Gene therapy
