Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease.

The article focuses on research which described the use of a machine-learning technique to identify small molecules that correct gene networks dysregulated in a human pluripotent stem cell disease model of an aortic heart valve disease. A targeted RNA-seq strategy assaying expression of 119 signature genes designed to map the gene network disrupted by N1 haploinsufficiency is described. Reduction of aortic valve disease in vivo by network-correcting molecules is discussed in detail.