miRagen’s miRNA Programs

miRNA expression profiling in a variety of tissue, cell and disease types has revealed a “miRNA signature” specific to those cell types or disease states. miRagen employees have identified miRNA signature expression patterns associated with pathological cardiac hypertrophy, heart failure (van Rooij et al. Proc. Natl. Acad. Sci., 2006.) and myocardial infarction in humans and mouse models of heart diease (van Rooij et al., Pro. Natl. Acad. Sci., 2008), as well as discovering miRNAs that are important in vascular (Xin et al Genes Dev., 2009) and skeletal muscle disease (Williams, Science, 2009, van Rooij, Dev Cell., 2009, Wang et al Dev Cell., 2008.). For example, it has been found that miR-206 is expressed only in skeletal muscle, miR-208 is found only in the heart (van Rooij et al. Science, 2007), and miR-126 is expressed specifically in endothelial cells lining the blood vessels (Wang et al. Dev Cell., 2008) .

Recent studies have uncovered profound and unexpected roles for microRNAs and their ability to control diverse aspects of disease. miRagen has shown in a knockout model for miR-208 that animals experience a loss of cardiac hypertrophic response compared to normal animals suggesting that hypertrophy can be modulated by altering miR-208 levels (van Rooij et al. Science, 2007). miRagen aims to use miRNA based discoveries to develop new therapies for different aspects of cardiovascular and muscle biology.