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The therapeutic potential of gene silencing by RNA interference has generated considerable interest and investment, with clinical trials now in progress in various disease areas. However, achieving target specificity remains a key challenge. Here, Jackson and Linsley discuss the off-target effects of small interfering RNAs and strategies aimed to mitigate them.
Despite an arsenal of antiepileptic drugs, ∼30% of patients with epilepsy are not free from seizures. This article reviews the emerging understanding of the molecular basis of epilepsy and experimental models of the disease, and how they might be used in the design of new therapeutics.
Although considerable efforts have been made to deliver drugs to particular tissues, little is known about targeting drugs to specific cellular compartments. By examining the fundamental principles of membrane trafficking and subcellular organization, the authors outline strategies to increase drug concentrations specifically in the relevant subcellular locations.
