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A number of compounds, derived from bacterial fermentation products, have been found to target subunits of the spliceosome and display anticancer properties. In this Review, Valcárcel and colleagues discuss the role of splicing in cancer and how insights into the mechanism of action of these bacterial compounds might lead to the development of novel antitumour drugs.
The market for drugs to treat wet age-related macular degeneration (AMD) is expected to more than double by 2016. This article investigates unmet needs and discusses current agents in the pipeline.
MicroRNAs (miRNAs) are involved in the regulation of gene expression and have been implicated in the pathology of several diseases. Here, van Rooij and Olson discuss the chemistry of current miRNA inhibitors and evaluate miRNAs as potential therapeutic targets for cardiovascular disorders.
Vemurafenib is a small-molecule inhibitor of the oncogenic BRAF kinase approved for the treatment of melanoma. Here, the scientists involved in the discovery of vemurafenib describe the underlying biology of BRAF, the technology used to identify vemurafenib and its clinical development milestones, along with future prospects based on lessons learned during its development.