Since microRNAs have functions in diverse biological pathways, altered expression or function of microRNAs give rise to numerous diseases including cancer and fibrosis, as well as CNS, metabolic and inflammatory disorders. The demonstration that a number of microRNAs are dysregulated in a particular disease phenotype has provided the rationale to use anti-miR and mimics technology to modulate aberrant microRNA function, thus restoring the balance of normal gene regulation inside the cell. This microRNA modulation strategy provides a unique approach to multiple diseases by utilizing oligonucleotides to target microRNAs that are altered in human disease.
Developing microRNA therapeutics requires an understanding of microRNA targets as well as the pathways they regulate. Since microRNAs regulate networks of genes, knowledge of links between microRNA targets and function becomes a challenge in network biology. Regulus scientists are developing novel methods and algorithms to monitor the activity of microRNA-modulating drugs and to focus on the functional consequences of gene network alterations.
microRNA Tissue Expression and Compound Delivery Mapping
Regulus has extensively mapped microRNA expression in different human and mouse tissues. In addition to tissue expression, it is essential to understand functional delivery of oligonucleotide compounds in different organs and tissues. Regulus scientists comprehensively mapped functional delivery of microRNA-modulating compounds in mouse tissues via monitoring of mRNA target modulation.
The combination of disease pathology, microRNA expression and functional delivery of chemically modified microRNA modulators enable us to discover novel therapeutic opportunities. Our target identification and validation approach combines our knowledge of microRNAs, their role in disease with high unmet need, translatable models and rigorous delivery mapping for our compounds.