Genetic mutations can cause numerous diseases. These alterations affect not only protein-coding genes but also regions that were until recently thought to be trivial in disease. In 'Bedside to Bench', David Salzman and Joanne Weidhaas examine a human study showing how a silent mutation impairs the binding of miR-196, increasing the risk for Crohn's disease. Therefore, alterations of miRNA target sites as pathogenic mechanism begs further investigation. miRNAs themselves can also be the root of disease. In 'Bench to Bedside', Carlo Croce peruses a study in vivo showing evidence of tumor addiction to miR-21 in a mouse model of cancer, which highlights the role of miRNAs as initiators of disease. Targeting these drivers will help to develop effective drugs.
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Mary Lou Quillen
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J.B.W. is co-inventor on several patents and is the founder of a company, miraDx.
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Salzman, D., Weidhaas, J. miRNAs in the spotlight: Making 'silent' mutations speak up. Nat Med 17, 934–935 (2011). https://doi.org/10.1038/nm0811-934
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DOI: https://doi.org/10.1038/nm0811-934
