Targeting Neurological Disease with siRNA

RNA interference (RNAi) is a mechanism that specifically inhibits gene expression via small RNAs such as small interfering RNA (siRNA) and microRNA (miRNA). It not only plays a pivotal role under physiological conditions as cell differentiation and development but it also permits a systematic research on gene function and the identification of potential drug targets. In neuroscientific research, RNAi technologies are broadly used to unravel gene function under physiological and pathological conditions. Moreover, RNAi has already been successfully employed as a therapeutic approach in different animal models of neurological disease in vivo. Here, we present an overview on RNAi-mediated gene silencing focusing on issues of design, delivery, and detrimental effects of siRNAs. We review the current use of RNAi in different models of neurological disease and give an outlook on the possibilities of implementation of this technology in clinical therapy.