RNA interference as a therapeutic strategy for treating CNS disorders

RNA interference (RNAi) controls gene silencing in most living organisms. The potential clinical applications of RNAi represent a strategy with unsurpassed selectivity, with the ability to target multiple disease-related genes, independent of their perceived drugability. The design of highly selective and efficacious small interfering (siRNAs) and short hairpin RNAs (shRNAs) has become routine, owing to significant progress in modeling and chemistry. RNAi significantly downregulates gene expression and function both in vitro and in vivo, including in the brain. This essay highlights recent findings and how the pharmaceutical industry intends to apply RNAi for the treatment neuropsychiatric and other diseases.

Section editors:David Sibley – National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, USAC. Anthony Altar – Psychiatric Genomics, Gaithersburg, USATheresa Branchek – Lundbeck Research, Paramus, USA