Abstract
When new protein structures are reported, it’s often claimed they could lead to the design of therapeutic agents that interact with the proteins more effectively. A new study on short interfering RNAs (siRNAs) actually demonstrates such a result. Last year, two research groups determined crystal structures of the human version of Argonaute—a protein in the RNA interference (RNAi) pathway that cells use to turn off target genes. siRNAs activate RNAi in cells by binding to messenger RNAs for targeted genes and marking them for destruction by Argonaute. siRNAs could thus be used therapeutically to turn off problematic genes related to various diseases. But designing siRNA drugs isn’t easy because they don’t readily enter cells, are broken down and excreted quickly, produce immune reactions, and cause side effects by inhibiting off-target genes. Nevertheless, several chemically modified siRNA drug candidates have entered human clinical trials. Dean J. Tantillo, Peter A. Beal, and ...
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