Science Spotlight: As miRNAs get Nobel Prize, targeting advances continue

The discovery of microRNAs, for which Victor Ambros and Gary Ruvkun won this year’s Nobel Prize in Physiology or Medicine, cracked open a world non-coding RNAs that has greatly expanded in the intervening years, but translating microRNAs to therapeutics, either as a modality or a target, has yet to take off. 

MicroRNAs (miRNAs), typically about 21-25 nucleotides in length, regulate gene expression by binding to complementary sequences on mRNAs and inhibiting their translation. The two Nobel laureates discovered miRNA in C. elegans, but it quickly became clear that miRNA are widespread among multicellular organisms, including humans...