Genetic diseases in the omics era

Abstract

Accurate quantification of gene expression at RNA and protein levels has provided invaluable insight into the mechanisms of biological processes in health and disease. The so-called “-omics” technologies (transcriptomics, proteomics, but also epigenomics, etc.) enabled researchers to detect expression changes specifically associated with given biological states and, more recently, to establish their dynamics at a single-cell resolution in stem cell-based models. In this issue of Molecular Therapy, Djeddi et al.