MicroRNA therapeutics: a new niche for antisense nucleic acids.
MicroRNA molecules (miRNAs) are naturally occurring triggers of the RNA-interference pathway. The first identified miRNA, lin-4, was discovered in Caenorhabditis elegans >20 years ago. What began as a curiosity in this model organism has expanded into almost every area of biology; there are now 326 confirmed miRNA genes in humans and the total is predicted to reach 1000. Each miRNA has the potential to regulate hundreds of mRNAs; therefore, there are likely to be few biological pathways not impacted by miRNA regulation. Recent evidence has suggested that miRNAs might be viable therapeutic targets for a wide range of diseases, including cancer. A recent article by Stoffel and colleagues has demonstrated remarkably effective inhibition of miRNAs in vivo, thus providing an entry point into the promising new arena of miRNA therapeutics.
Hammond
Scott M
SM
Department of Cell and Developmental Biology, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC 27599, USA. Hammond@med.unc.edu
D000818:Animals; D017173:Caenorhabditis elegans / Q000235:genetics; D005786:Gene Expression Regulation; D006801:Humans; D035683:MicroRNAs / Q000037:antagonists & inhibitors / Q000378:metabolism / Q000627:therapeutic use; D016376:Oligonucleotides, Antisense / Q000378:metabolism*; D034622:RNA Interference
D035683:MicroRNAs; D016376:Oligonucleotides, Antisense