Investigational Compounds

MicroRNAs (miRNAs) are a class of  non-coding RNA molecules that play an essential role in the regulation of gene expression at post-transcriptional levels by binding to target mRNAs and inhibiting protein synthesis. The most common way to inhibit microRNAs is the administration of synthetic single-stranded oligonucleotides (called antimiRs) with a sequence complementary to that of the miRNA to be inhibited. These antimiRs bind to the target miRNA by blocking or degrading it. To improve the binding affinity, stability and bio-distribution of the antimRs, much progress has been made in the generation of new chemical modifications in the nucleotides that make them up.