EMBO Workshop — Non-coding RNA medicine

The discovery of myriad noncoding RNAs (ncRNAs) over the past decade has revealed a universe of new and exciting therapeutic opportunities. Many thousands of microRNAs, piRNAs, circular RNAs, long noncoding RNAs, and others are expressed across healthy and diseased human tissues and play key functional roles in pathogenic states and processes. Their regulatory roles, cell-type-specificity, and vast numbers confer on ncRNAs unique advantages as therapeutic targets. Rapid advances in clinical transcriptomics, oligonucleotide therapeutics, and RNA-directed small molecules, open the possibility for rapid, economical, and personalised ncRNA-directed therapies. Reaching this goal will require overcoming key challenges, including low evolutionary conservation in model species, poorly-characterised molecular mechanisms, finding hits amongst the vast ncRNA transcriptome, and effectively drugging targets in vivo.