The Emerging Therapeutic Role of RNA Interference in Disorders of the Central Nervous System

Several challenges have been encountered in the attempt to apply RNA interference (RNAi) to the treatment of disease, including lack of effective delivery, off-target responses, and instability of reagents. The treatment of neuropsychiatric disorders poses additional obstacles, given that drugs have only limited access to the central nervous system (CNS) and that the neurons are in a postmitotic state. Despite these inherent limitations, much progress has been made toward attaining a better understanding of diseases of the CNS, and the development of effective treatments appears to be a distinct possibility in the near future.