A06: Decoding the regulatory network of RBM20

A06 pursues a novel strategy to treat an inherited form of dilated cardiomyopathy caused by mutations in the gene RBM20. DCM patients usually harbor one defective copy of RBM20 while the other one is still intact (haploinsufficieny). Upregulation of the “healthy” allele presents a pathway to restore RBM20 activity. By systematically manipulating the expression of thousands of genes, and analyzing their impact on the expression of RBM20 protein, we seek to uncover cellular pathways that can be targeted by drugs that modulate RBM20 expression and rescue the effects of the disease-causing mutations.