RNA-targeted therapeutics represent a promising new class of agents for drug discovery, driven by advances in structural biology and screening methods.
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RNA-targeted ligands merit further preclinical investment; leaves open clinical translation pending prospective validation.
Structured regions of viral, bacterial and human RNA are attractive targets for drug discovery. Interest is fuelled by the revelation, from nuclear magnetic resonance (NMR) and X-ray crystallography, that RNA has a structural complexity similar to proteins, and also by recent development of novel RNA-focused screening methods. However, few examples of RNA-targeted ligands have been reported to date. Analysis of these compounds, and their target specificity, suggests that considerable promise exists for the future development of new classes of therapeutic agents.
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Swayze et al. (2002) studied this question.
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