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Mullen and Scheffer Article provide an elegant update of the genetics of sodium channelopathies and epilepsy. Recently-developed animal models of sodium channel–gene SCN1A mutants recapitulate the human disease. Their review illustrates the key role basic science plays in the development of targeted novel therapies and that ultimately will lead to the prevention of these serious disorders.
Yokota et al Article provide new insights into antisense oligonucleotides, short nucleic acid sequences designed for use as small-molecule drugs that bind to specific mRNA sequences and alter mRNA-splicing patterns or inhibit protein synthesis. Impressive preclinical and clinical data using antisense have been shown to increase dystrophin production in Duchenne muscular dystrophy. Clearly, this technology will have a major effect as therapy for genetic neurological disease in the near future.
This Month in Archives of Neurology. Arch Neurol. 2009;66(1):17–18. doi:https://doi.org/10.1001/archneurol.2008.543
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