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Isothermal titration calorimetry can be used to obtain thermodynamic data on the binding of compounds to protein targets. Ladbury and colleagues propose that these data — particularly the change in enthalpy — could be a valuable addition to established tools for selecting compounds in lead discovery and for aiding lead optimization.
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.
The complement system has long been known to have a pathogenic role in various disorders, but the therapeutic potential of modulating complement responses has only recently been recognized. In this article, Wagner and Frank review those diseases associated with complement activation and deregulation and focus on promising therapeutic strategies and agents that are currently in the clinic.
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.
